X4 Pharmaceuticals is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing novel therapeutics for primary immune deficiencies and cancer. Its lead drug candidate, mavorixafor, is an oral small molecule antagonist of the chemokine receptor CXCR4 and is currently undergoing various clinical trials, including a Phase III trial for patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome, as well as trials for severe congenital neutropenia and clear cell renal cell carcinoma. The company is also advancing X4P-002, which targets glioblastoma multiforme, and X4P-003, aimed at treating primary immune deficiencies. Additionally, X4 Pharmaceuticals has a collaboration with Abbisko Therapeutics to explore mavorixafor in combination with checkpoint inhibitors for oncology applications. Founded in 2014, X4 Pharmaceuticals was previously known as Arsanis, Inc. before rebranding in 2019.
Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an investigational therapy. The company's lead product, Haduvio, is an oral extended-release formulation of nalbuphine currently undergoing Phase IIb/III clinical trials. Trevi Therapeutics is focused on addressing serious neurologically mediated conditions, including chronic pruritus, chronic cough associated with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in Parkinson's disease patients. The ongoing PRISM trial evaluates Haduvio's effectiveness in treating severe pruritus related to prurigo nodularis, highlighting its potential in managing conditions linked by a common pathophysiology involving opioid receptors in the nervous system. Founded in 2011, Trevi Therapeutics aims to provide innovative therapeutic solutions for patients with significant unmet medical needs.
Sorriso Pharmaceuticals is a biotechnology company based in San Diego, California, founded in 2020. The company focuses on developing disease-modifying antibodies aimed at treating inflammatory diseases such as Crohn's disease and ulcerative colitis. Sorriso's innovative platform produces potent antibodies that can be delivered orally, ensuring they remain effective throughout the intestinal system. This approach aims to provide clinical benefits comparable to traditional injectable antibody therapeutics, thereby alleviating the treatment burden for patients who typically rely on injections for their care. By advancing its pipeline of oral therapies, Sorriso Pharmaceuticals seeks to improve patient outcomes and enhance the management of inflammatory diseases.
Recludix pharma is a developing platform to discover potent and selective inhibitors of challenging protein targets. The platform approach integrates custom generated DNA-encoded libraries, massively parallel determination of structure-activity relationships, and a proprietary screening tool to ensure selectivity.
858 Therapeutics
Series A in 2021
858 Therapeutics is a stealth company specializing in the area of healthcare services, pharmaceutical, and biotechnology. The company was founded in 2019 and is headquartered in San Diego, California, USA.
Allay Therapeutics offers analgesic products for post-surgical pain management and recuperation. The company was founded in 2017 and is headquartered in Menlo Park, California, United States.
Stablix Therapeutics is a biotechnology company pioneering the field of Targeted Protein Stabilization (TPS). The company’s resorted platform generates heterobifunctional small molecules (RESTORACS) that recruit deubiquitinase enzymes to remove ubiquitin from targeted proteins and consequently stabilize or increase target protein levels and activity. Stablix initially is leveraging the platform to develop programs to treat rare diseases, cancer, and immunological disorders.
Monte Rosa Therapeutics is a biotechnology company based in Basel, Switzerland, that specializes in developing innovative cancer therapeutics through the modulation of protein degradation pathways. The company focuses on molecular glue degraders (MGDs), a class of small molecule drugs that utilize the body's natural mechanisms for protein degradation to selectively target and degrade proteins relevant to cancer treatment. Monte Rosa employs its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, to identify and develop target proteins for degradation. With a library of over 50,000 MGD molecules, its leading product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing potential for treating MYC-driven tumors. The company is committed to advancing its pipeline of novel therapeutics to address unmet medical needs in oncology.
Satsuma Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraines. The company's primary product candidate, STS101, is a drug-device combination featuring a proprietary dry-powder formulation of dihydroergotamine mesylate. This medication is designed for self-administration using a pre-filled, single-use nasal delivery device. Currently, STS101 is undergoing Phase III clinical trials, marking a significant step in its development process. Founded in 2016 and based in South San Francisco, California, Satsuma Pharmaceuticals aims to provide effective solutions for individuals suffering from acute migraine attacks.
Sagimet Biosciences is a biotechnology company that focuses on developing novel therapeutics to treat important diseases such as liver disease NASH and specific cancers, with a focus on targeting dysfunctional metabolic pathways. It was founded in 2007 and headquartered in San Mateo, California.
Reneo Pharmaceuticals develops therapies for patients with rare genetic mitochondrial diseases, which impact cellular metabolism and energy production. Founded in 2014 and based in San Diego, California, the company is focused on creating treatments that enhance mitochondrial function, thereby improving muscle health and overall quality of life for affected individuals. Its lead product candidate, REN001, is an oral medication designed to modulate genes essential for energy metabolism and the production of adenosine triphosphate (ATP), the primary energy source for cellular processes. By addressing the underlying metabolic deficiencies in these patients, Reneo aims to prevent muscle injury, weakness, and wasting, ultimately enhancing daily functioning for those with these orphan diseases.
Resilience is a biopharmaceutical manufacturing and technology company based in La Jolla, California, established in 2020. It aims to enhance access to complex medicines and safeguard biopharmaceutical supply chains. The company specializes in end-to-end manufacturing and development solutions across various therapeutic modalities, including cell and gene therapies, viral vectors, vaccines, and proteins. Resilience provides customized manufacturing capabilities that cater to the diverse needs of biopharmaceuticals at all stages of the drug development process, from pre-clinical development to commercial supply. With a focus on developing new manufacturing technologies, Resilience ensures high-quality and regulatory-compliant processes for complex and novel medicines.
Monte Rosa Therapeutics is a biotechnology company based in Basel, Switzerland, that specializes in developing innovative cancer therapeutics through the modulation of protein degradation pathways. The company focuses on molecular glue degraders (MGDs), a class of small molecule drugs that utilize the body's natural mechanisms for protein degradation to selectively target and degrade proteins relevant to cancer treatment. Monte Rosa employs its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, to identify and develop target proteins for degradation. With a library of over 50,000 MGD molecules, its leading product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing potential for treating MYC-driven tumors. The company is committed to advancing its pipeline of novel therapeutics to address unmet medical needs in oncology.
Polyneuron Pharmaceuticals AG is focused on developing innovative therapeutics for the treatment of antibody-mediated autoimmune disorders. The company is working on several drug candidates, including PN-1007 for anti-MAG neuropathy, PN-1018 for multifocal motor neuropathy, and PN-032 and PN-056 for ABO-incompatible transplantation. Polyneuron utilizes a platform technology called Antibody-Catch, which is based on biodegradable glycopolymers. This technology selectively targets and eliminates pathological autoantibodies while preserving the integrity of the rest of the immune system. Founded in 2014 and headquartered in Basel, Switzerland, Polyneuron aims to provide safe and effective treatment options for patients suffering from autoimmune diseases affecting the peripheral nervous system.
MBX Biosciences, Inc. is a biotechnology company based in Carmel, Indiana, specializing in the development of therapeutics for rare endocrine disorders. Founded in 2018, the company aims to address unmet medical needs by discovering and commercializing innovative treatments for endocrine diseases that affect hormonal regulation in the body. MBX Biosciences focuses on creating first-in-class therapies, particularly utilizing peptides to target rare genetic conditions related to glandular hormone secretion.
Monte Rosa Therapeutics is a biotechnology company based in Basel, Switzerland, that specializes in developing innovative cancer therapeutics through the modulation of protein degradation pathways. The company focuses on molecular glue degraders (MGDs), a class of small molecule drugs that utilize the body's natural mechanisms for protein degradation to selectively target and degrade proteins relevant to cancer treatment. Monte Rosa employs its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, to identify and develop target proteins for degradation. With a library of over 50,000 MGD molecules, its leading product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing potential for treating MYC-driven tumors. The company is committed to advancing its pipeline of novel therapeutics to address unmet medical needs in oncology.
Amplyx Pharmaceuticals is a small molecule drug development company focused on creating innovative therapies for life-threatening infections, particularly in patients with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in developing oral and injectable anti-infective drugs, including antifungal agents aimed at treating invasive fungal infections caused by pathogens such as Candida and Aspergillus. Amplyx employs a unique platform approach that enhances the efficacy and reduces the toxicity of existing drugs by combining them with additional small molecules. This strategy addresses critical treatment needs in hospital settings and provides effective therapy options for patients after discharge, particularly those undergoing chemotherapy and other immunosuppressive treatments.
Inventiva S.A. is a biopharmaceutical company based in Daix, France, specializing in the development of oral small molecule therapies targeting significant unmet medical needs in fibrosis, lysosomal storage disorders, and oncology. The company's lead candidate, Lanifibranor, is currently in Phase IIb clinical trials for the treatment of nonalcoholic steatohepatitis. Additionally, Inventiva is advancing Odiparcil, in Phase IIa trials for mucopolysaccharidosis type VI, and has several preclinical programs, including potential treatments for malignant mesothelioma and lung cancer. The company's research and development efforts are supported by a proprietary library of molecules and a team experienced in targeting nuclear receptors and transcription factors. Inventiva has established partnerships with prominent organizations, including the Institut Curie for oncology research and AbbVie for autoimmune disease treatments. Founded in 2011, the company continues to focus on innovative solutions for complex medical conditions.
ERYTECH Pharma S.A. is a clinical-stage biopharmaceutical company based in Lyon, France, founded in 2004. The company specializes in the development of red blood cell-based therapeutics aimed at treating cancer and orphan diseases. Its lead product candidate, eryaspase, is currently undergoing Phase III clinical trials for second-line pancreatic cancer and Phase II trials for triple-negative breast cancer. Additionally, ERYTECH is developing erymethionase, a preclinical candidate targeting methionine-dependent cancers, which consists of methionine-gamma-lyase encapsulated in red blood cells. The company collaborates with research institutions such as the Fox Chase Cancer Center and Queen’s University of Canada to enhance the preclinical development of its products for conditions like homocystinuria and arginase-1 deficiency, respectively. Through its innovative approach to encapsulating therapeutic molecules, ERYTECH Pharma aims to improve the efficacy and safety of its treatments.
Inventiva S.A. is a biopharmaceutical company based in Daix, France, specializing in the development of oral small molecule therapies targeting significant unmet medical needs in fibrosis, lysosomal storage disorders, and oncology. The company's lead candidate, Lanifibranor, is currently in Phase IIb clinical trials for the treatment of nonalcoholic steatohepatitis. Additionally, Inventiva is advancing Odiparcil, in Phase IIa trials for mucopolysaccharidosis type VI, and has several preclinical programs, including potential treatments for malignant mesothelioma and lung cancer. The company's research and development efforts are supported by a proprietary library of molecules and a team experienced in targeting nuclear receptors and transcription factors. Inventiva has established partnerships with prominent organizations, including the Institut Curie for oncology research and AbbVie for autoimmune disease treatments. Founded in 2011, the company continues to focus on innovative solutions for complex medical conditions.
Sagimet Biosciences is a biotechnology company that focuses on developing novel therapeutics to treat important diseases such as liver disease NASH and specific cancers, with a focus on targeting dysfunctional metabolic pathways. It was founded in 2007 and headquartered in San Mateo, California.
Reneo Pharmaceuticals develops therapies for patients with rare genetic mitochondrial diseases, which impact cellular metabolism and energy production. Founded in 2014 and based in San Diego, California, the company is focused on creating treatments that enhance mitochondrial function, thereby improving muscle health and overall quality of life for affected individuals. Its lead product candidate, REN001, is an oral medication designed to modulate genes essential for energy metabolism and the production of adenosine triphosphate (ATP), the primary energy source for cellular processes. By addressing the underlying metabolic deficiencies in these patients, Reneo aims to prevent muscle injury, weakness, and wasting, ultimately enhancing daily functioning for those with these orphan diseases.
Polyneuron Pharmaceuticals AG is focused on developing innovative therapeutics for the treatment of antibody-mediated autoimmune disorders. The company is working on several drug candidates, including PN-1007 for anti-MAG neuropathy, PN-1018 for multifocal motor neuropathy, and PN-032 and PN-056 for ABO-incompatible transplantation. Polyneuron utilizes a platform technology called Antibody-Catch, which is based on biodegradable glycopolymers. This technology selectively targets and eliminates pathological autoantibodies while preserving the integrity of the rest of the immune system. Founded in 2014 and headquartered in Basel, Switzerland, Polyneuron aims to provide safe and effective treatment options for patients suffering from autoimmune diseases affecting the peripheral nervous system.
Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing innovative therapeutics for patients with rare genetic disorders of hemoglobin, particularly sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing IMR-687, a once-daily oral therapy designed to be a potent small molecule inhibitor of PDE9. This therapeutic aims to address the significant health challenges associated with these conditions, which can lead to reduced healthy red blood cells and various complications. Imara was established following a collaboration between Cydan Development and H. Lundbeck A/S, with initial funding from notable life science investors.
Oyster Point Pharma, Inc. is a clinical-stage biopharmaceutical company based in Princeton, New Jersey, that specializes in the discovery, development, and commercialization of innovative therapies for ocular surface diseases. Founded in 2015, the company is primarily focused on its lead product candidate, OC-01, a nicotinic acetylcholine receptor agonist currently undergoing Phase III clinical trials for the treatment of dry eye disease. This condition, which affects over 30 million Americans, is characterized by a chronic lack of sufficient tears, leading to discomfort and potential vision problems. OC-01 is developed as a nasal spray and aims to restore tear film homeostasis by activating the trigeminal parasympathetic pathway, thereby stimulating natural tear production. Additionally, the company is exploring the use of OC-01 for neurotrophic keratitis, further expanding its therapeutic potential in ocular health.
Tiburio Therapeutics is a developer of drug compounds intended to treat tumors and endocrine diseases.The company's compounds aid in the treatment of non-functioning pituitary adenoma using a dopamine-somatostatin chimeric molecule that has the potential to shrink or halt tumor growth, providing patients with effective treatment for rare neuroendocrine tumors and endocrine diseases.
Curzion Pharmaceuticals
Venture Round in 2019
Curzion Pharmaceuticals is a privately held development-stage biopharma company, and its development-stage oral selective lysophosphatidic acid 1 receptor (LPAR1) antagonist, CZN001 (renamed HZN-825).
Allay Therapeutics offers analgesic products for post-surgical pain management and recuperation. The company was founded in 2017 and is headquartered in Menlo Park, California, United States.
Xenon Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Burnaby, Canada, focused on developing innovative therapeutics for neurological disorders. The company's clinical pipeline includes several product candidates targeting epilepsy, such as XEN496 and XEN1101, both Kv7 potassium channel modulators currently in Phase II trials, and XEN901, a selective Nav1.6 sodium channel inhibitor in Phase I trials. Additionally, XEN007, a central nervous system-acting calcium channel modulator, is also in Phase I trials. Xenon utilizes a proprietary discovery platform, known as Extreme Genetics, to identify genetic insights from families with severe phenotypes, enabling the development of novel medicines for both orphan and more common diseases. The company has established a collaboration with Neurocrine Biosciences to advance first-in-class treatments for epilepsy, highlighting its commitment to addressing unmet medical needs in the field of neurology.
Comprehensive Pharmacy Services, Inc. (CPS) provides a broad range of pharmacy support services to over 800 hospitals and healthcare facilities across the United States. Founded in 1971 and headquartered in Memphis, Tennessee, CPS specializes in inpatient hospital pharmacy services, clinical consulting, and operational assessments. The company addresses various challenges faced by healthcare providers, including high drug costs, specialty pharmacy management, and operational efficiency. CPS also offers consulting solutions that encompass compliance, medication safety, sterile compounding, and pharmacy analytics. Through its extensive service offerings, CPS aims to enhance the quality and effectiveness of pharmacy operations within healthcare settings.
Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an investigational therapy. The company's lead product, Haduvio, is an oral extended-release formulation of nalbuphine currently undergoing Phase IIb/III clinical trials. Trevi Therapeutics is focused on addressing serious neurologically mediated conditions, including chronic pruritus, chronic cough associated with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in Parkinson's disease patients. The ongoing PRISM trial evaluates Haduvio's effectiveness in treating severe pruritus related to prurigo nodularis, highlighting its potential in managing conditions linked by a common pathophysiology involving opioid receptors in the nervous system. Founded in 2011, Trevi Therapeutics aims to provide innovative therapeutic solutions for patients with significant unmet medical needs.
Mirum Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of therapies for debilitating liver diseases. It is involved in the development of Maralixibat for the treatment of pediatric patients with progressive familial intrahepatic cholestasis, or PFIC, and Alagille syndrome, or ALGS. The company is also involved in the development of volixibat for the treatment of adult patients with cholestatic liver diseases.
Qpex Biopharma is a San Diego-based biopharmaceutical company founded in 2018, focused on developing innovative therapies to address the growing challenge of antimicrobial resistance. The company specializes in creating a pipeline of antibiotic drugs aimed at treating infectious diseases in both inpatient and outpatient settings. With a strong emphasis on the discovery, development, and regulatory approval of anti-infective medicines, Qpex Biopharma leverages its expertise and experience in collaboration with public and private partnerships to meet critical medical needs. The company's mission is to provide effective solutions for healthcare professionals facing the complexities of resistant infections, thereby enhancing treatment options for patients.
PhaseBio Pharmaceuticals is a clinical-stage biopharmaceutical company based in Malvern, Pennsylvania, focused on developing innovative therapies for cardiopulmonary diseases. Its lead product candidate, PB2452, is a reversal agent for the antiplatelet drug ticagrelor and is currently undergoing Phase III clinical trials for patients experiencing uncontrolled bleeding or requiring urgent surgery. The company is also advancing PB1046, a fusion protein aimed at treating pulmonary arterial hypertension, which is in Phase IIb clinical trials. Additionally, PhaseBio is developing PB6440, an oral agent intended for the treatment of resistant hypertension. Founded in 2002, the company leverages proprietary recombinant biopolymers to enhance the stability and efficacy of its drug candidates.
Atterocor, Inc. focuses on research and development of therapeutics for adrenal disease. Atterocor, Inc. was incorporated in 2012 and is based in Ann Arbor, Michigan.
Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an investigational therapy. The company's lead product, Haduvio, is an oral extended-release formulation of nalbuphine currently undergoing Phase IIb/III clinical trials. Trevi Therapeutics is focused on addressing serious neurologically mediated conditions, including chronic pruritus, chronic cough associated with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in Parkinson's disease patients. The ongoing PRISM trial evaluates Haduvio's effectiveness in treating severe pruritus related to prurigo nodularis, highlighting its potential in managing conditions linked by a common pathophysiology involving opioid receptors in the nervous system. Founded in 2011, Trevi Therapeutics aims to provide innovative therapeutic solutions for patients with significant unmet medical needs.
Metacrine, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapies for liver and gastrointestinal diseases. The company is advancing its lead programs, which target the farnesoid X receptor (FXR), a key drug target in these conditions. Currently, MET409 is undergoing a Phase Ib proof-of-concept clinical trial for patients with non-alcoholic steatohepatitis, while MET642 is in a Phase 1 clinical trial for the same patient population. In addition to its FXR-focused programs, Metacrine is exploring a pipeline of novel drug candidates through its internal research and development capabilities. The company also collaborates with Novo Nordisk A/S to further research related to fibroblast growth factor 1. Metacrine was founded in 2014 and is committed to addressing unmet medical needs in liver and gastrointestinal health.
CareZone is a healthcare platform focused on simplifying personal health management and pharmacy delivery services. It offers tools that automate prescription refill reminders, track medical appointments, and provide controlled access to health information for family members and caregivers. This integrated approach allows users to organize their health data and coordinate care effectively from a single interface, making it easier for individuals and families to manage their healthcare needs.
MEI Pharma, Inc. (Nasdaq: MEIP) is a San Diego-based oncology company focused on the clinical development of novel therapies for cancer. The Company’s clinical development pipeline includes lead drug candidate Pracinostat, a potential best-in-class, oral histone deacetylase (HDAC) inhibitor. Pracinostat has been tested in more than 150 patients in multiple Phase I and exploratory Phase II clinical trials, including advanced hematologic malignancies such as myelodysplastic syndrome, acute myeloid leukemia and myelofibrosis. The Company expects to initiate a randomized Phase II trial of Pracinostat in combination with standard-of-care in at least one hematologic malignancy toward the middle of 2013. In addition, MEI Pharma is developing two drug candidates derived from its isoflavone-based technology platform, ME-143 and ME-344. Results from a Phase I trial of intravenous ME-143 in heavily treated patients with solid refractory tumors were presented at the American Society of Clinical Oncology Annual Meeting in June 2012. A Phase I clinical trial of intravenous ME-344 in patients with solid refractory tumors is ongoing.
Liquidia Technologies is a late-stage clinical biopharmaceutical company based in Morrisville, North Carolina, that specializes in the development and commercialization of human therapeutics. The company utilizes its proprietary PRINT technology, a particle engineering platform that allows for the precise production of uniform drug particles, aimed at improving the safety, efficacy, and performance of various therapies. Liquidia is currently advancing two key product candidates: LIQ861 for the treatment of pulmonary arterial hypertension and LIQ865 for addressing local post-operative pain. In addition to its internal pipeline, Liquidia collaborates with leading pharmaceutical companies to leverage its PRINT technology across multiple therapeutic areas and drug types. The company also offers strategy and commercialization services for rare disease pharmaceutical products, including a generic formulation of treprostinil for pulmonary arterial hypertension. Founded in 2004, Liquidia Technologies continues to focus on transforming patient care through innovative therapeutic solutions.
Xoc utilizes state-of-the-art receptor characterization technology and cutting-edge medicinal chemistry to identify product candidates and develop novel therapies to meet the goal of providing improved safety and efficacy for patients. Xoc manages the clinical and regulatory risk in our development programs by leveraging the extensive pharmacological history of compounds similar to their product candidates.
The name “Xoc” is derived from a term in the Mayan language and is pronounced “shock”.
Centrexion Therapeutics Corporation is a late clinical-stage biopharmaceutical company dedicated to developing novel, non-opioid, and non-addictive therapies for chronic pain management. Founded in 2013 and based in Boston, Massachusetts, the company’s product pipeline includes CNTX-4975, currently undergoing Phase III trials for knee osteoarthritis pain and Phase II trials for Morton’s neuroma and canine osteoarthritis. Additionally, CNTX-0290 is in Phase I trials for various chronic pain conditions, while CNTX-6970 targets inflammatory pain and CNTX-2022, a high-concentration topical gel formulation of lidocaine, is also in Phase I trials for superficial musculoskeletal pain and related conditions. Furthermore, CNTX-6016 is a pre-clinical candidate aimed at treating chronic neuropathic pain. Centrexion Therapeutics is committed to addressing the unmet medical needs in chronic pain treatment by focusing on safety and efficacy.
CTI BioPharma Corp. a biopharmaceutical company, develops, acquires, and commercializes oncology products for cancer treatment. Its development portfolio includes Pixantrone, a phase III trial product, for non-Hodgkin's lymphoma; Brostallicin, which is in first-line Phase II study for the treatment of sarcoma; and OPAXIO, a chemotherapeutic agent for the potential treatment of non-small cell lung, ovarian, and other cancers. It has collaboration and licensing arrangements with Novartis International Pharmaceutical, Ltd.; and PG-TXL Company, L.P. The company was founded in 1991 and is headquartered in Seattle, Washington.
Allakos Inc. is a clinical-stage biopharmaceutical company based in Redwood City, California, founded in 2012. The company specializes in developing antibody-based therapeutics aimed at treating various diseases linked to dysregulation of the T-helper type 2 immune response, including allergic diseases, inflammation, and conditions characterized by excessive inflammatory cell production. Its lead therapeutic candidate, antolimab (AK002), targets conditions such as eosinophilic gastritis, eosinophilic gastroenteritis, chronic urticaria, indolent systemic mastocytosis, and severe allergic conjunctivitis. Additionally, Allakos is advancing another program, AK006, which targets the Siglec-6 receptor on mast cells to modulate their activity and reduce excessive activation. Through these initiatives, Allakos aims to address significant unmet medical needs in immunological health.
Metacrine, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapies for liver and gastrointestinal diseases. The company is advancing its lead programs, which target the farnesoid X receptor (FXR), a key drug target in these conditions. Currently, MET409 is undergoing a Phase Ib proof-of-concept clinical trial for patients with non-alcoholic steatohepatitis, while MET642 is in a Phase 1 clinical trial for the same patient population. In addition to its FXR-focused programs, Metacrine is exploring a pipeline of novel drug candidates through its internal research and development capabilities. The company also collaborates with Novo Nordisk A/S to further research related to fibroblast growth factor 1. Metacrine was founded in 2014 and is committed to addressing unmet medical needs in liver and gastrointestinal health.
Oyster Point Pharma, Inc. is a clinical-stage biopharmaceutical company based in Princeton, New Jersey, that specializes in the discovery, development, and commercialization of innovative therapies for ocular surface diseases. Founded in 2015, the company is primarily focused on its lead product candidate, OC-01, a nicotinic acetylcholine receptor agonist currently undergoing Phase III clinical trials for the treatment of dry eye disease. This condition, which affects over 30 million Americans, is characterized by a chronic lack of sufficient tears, leading to discomfort and potential vision problems. OC-01 is developed as a nasal spray and aims to restore tear film homeostasis by activating the trigeminal parasympathetic pathway, thereby stimulating natural tear production. Additionally, the company is exploring the use of OC-01 for neurotrophic keratitis, further expanding its therapeutic potential in ocular health.
ObsEva SA is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, dedicated to developing innovative therapeutics for women's reproductive health and pregnancy. Founded in 2012 by Ernest Loumaye and André Chollet, the company focuses on addressing serious conditions such as endometriosis, heavy menstrual bleeding, and preterm labor. Its key products include Linzagolix, an oral gonadotropin-releasing hormone receptor antagonist aimed at treating pain from endometriosis and uterine fibroids, and OBE022, a selective prostaglandin F2α receptor antagonist designed for once-daily treatment of preterm labor. Additionally, ObsEva is advancing Nolasiban, an oral oxytocin receptor antagonist intended to enhance clinical pregnancy and live birth rates in women undergoing in vitro fertilization. The company operates primarily in Switzerland and is committed to improving outcomes for women facing reproductive health challenges.
Amplyx Pharmaceuticals is a small molecule drug development company focused on creating innovative therapies for life-threatening infections, particularly in patients with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in developing oral and injectable anti-infective drugs, including antifungal agents aimed at treating invasive fungal infections caused by pathogens such as Candida and Aspergillus. Amplyx employs a unique platform approach that enhances the efficacy and reduces the toxicity of existing drugs by combining them with additional small molecules. This strategy addresses critical treatment needs in hospital settings and provides effective therapy options for patients after discharge, particularly those undergoing chemotherapy and other immunosuppressive treatments.
Complexa, Inc. is a biopharmaceutical company based in Berwyn, Pennsylvania, focused on developing innovative treatments for severe diseases involving fibrosis and inflammation. Founded in 2008, Complexa specializes in the research and development of endogenous human cell signaling technologies, particularly through the synthesis of endogenous nitro-fatty acids. Their lead compound, CXA-10, is an oral nitrated fatty acid being developed for the treatment of focal segmental glomerulosclerosis and pulmonary arterial hypertension. The company aims to enhance existing anti-inflammatory and metabolic signaling pathways to promote recovery from acute and chronic tissue injuries and diseases, targeting a range of inflammatory, fibrotic, and central nervous system disorders.
Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an investigational therapy. The company's lead product, Haduvio, is an oral extended-release formulation of nalbuphine currently undergoing Phase IIb/III clinical trials. Trevi Therapeutics is focused on addressing serious neurologically mediated conditions, including chronic pruritus, chronic cough associated with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in Parkinson's disease patients. The ongoing PRISM trial evaluates Haduvio's effectiveness in treating severe pruritus related to prurigo nodularis, highlighting its potential in managing conditions linked by a common pathophysiology involving opioid receptors in the nervous system. Founded in 2011, Trevi Therapeutics aims to provide innovative therapeutic solutions for patients with significant unmet medical needs.
OrphoMed, Inc. develops therapies for irritable bowel syndrome with diarrhea (IBS-D), acute and chronic pain, and cystic fibrosis. Its pipeline includes ORP-101, a chemical entity designed to create a stable molecule and to confer partial μ agonist and full κ-antagonist receptor pharmacology; ORP-105, a non-addictive, centrally-acting, non-opiate analgesic; and ORP-110, a metabolically-stable, non-depletory cystic fibrosis transmembrane regulator potentiator. OrphoMed, Inc. was incorporated in 2015 and is based in San Francisco, California.
Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is an oral therapy currently in Phase I/IIa clinical trials for the treatment of phenylketonuria. Additionally, Synlogic is developing SYNB1891, an intratumoral synthetic biotic medicine that is undergoing Phase I clinical trials for the treatment of solid tumors and lymphoma. The company is also exploring synthetic biotics for more common conditions, including inflammatory bowel disease and secondary hyperoxaluria. Synlogic has established a collaboration with Ginkgo Bioworks to advance its development of these innovative therapies.
Addex Therapeutics Ltd is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, dedicated to the discovery and development of innovative small-molecule pharmaceutical products aimed at treating central nervous system (CNS) disorders. The company specializes in creating oral allosteric modulators that target G-protein coupled receptors, which are crucial for therapeutic interventions. Its leading programs include Dipraglurant, designed for treating levodopa-induced dyskinesia and dystonia in Parkinson’s disease, ADX71149 for epilepsy and other undisclosed CNS disorders, and a GABAB PAM aimed at addressing addiction. Addex Therapeutics has established a collaboration with Janssen Pharmaceuticals to advance the development of mGluR2PAM compounds for human health applications. Founded in 2002, the company has evolved its focus and pipeline based on a pioneering drug discovery platform that emphasizes allosteric modulation.
Rhythm Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing peptide therapeutics for the treatment of rare genetic disorders, particularly those leading to life-threatening metabolic conditions. Its primary product candidate, setmelanotide, is a first-in-class melanocortin-4 receptor agonist currently undergoing Phase III clinical trials for obesity related to pro-opiomelanocortin and leptin receptor deficiencies, as well as Bardet-Biedl and Alström syndromes. Additionally, setmelanotide is in Phase II trials for various other genetic obesity disorders. The company is also advancing RM-853, an orally available ghrelin O-acyltransferase inhibitor, in preclinical development for Prader-Willi syndrome. Founded in 2008 and headquartered in Boston, Massachusetts, Rhythm Pharmaceuticals aims to address significant unmet medical needs in the realm of genetic metabolic disorders through innovative therapies.
Tarveda Therapeutics is focused on developing innovative cancer therapies specifically for solid tumors. The company specializes in miniature drug conjugates, with its lead candidate, PEN-866, targeting activated Heat Shock Protein 90 (HSP90) to deliver a potent topoisomerase 1 inhibitor, SN38, directly to tumor cells. Additionally, Tarveda is advancing another drug conjugate aimed at treating patients with neuroendocrine tumors and small cell lung cancer that express somatostatin receptor 2 (SSTR2). By leveraging its targeted drug delivery platform, Tarveda Therapeutics aims to improve treatment outcomes for patients with difficult-to-treat cancers while reducing potential side effects. Founded in 2011 and based in Watertown, Massachusetts, the company was previously known as Blend Therapeutics until its rebranding in 2016.
Addex Therapeutics Ltd is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, dedicated to the discovery and development of innovative small-molecule pharmaceutical products aimed at treating central nervous system (CNS) disorders. The company specializes in creating oral allosteric modulators that target G-protein coupled receptors, which are crucial for therapeutic interventions. Its leading programs include Dipraglurant, designed for treating levodopa-induced dyskinesia and dystonia in Parkinson’s disease, ADX71149 for epilepsy and other undisclosed CNS disorders, and a GABAB PAM aimed at addressing addiction. Addex Therapeutics has established a collaboration with Janssen Pharmaceuticals to advance the development of mGluR2PAM compounds for human health applications. Founded in 2002, the company has evolved its focus and pipeline based on a pioneering drug discovery platform that emphasizes allosteric modulation.
Trillium Therapeutics Inc. is a clinical-stage immuno-oncology company based in Mississauga, Canada, focused on developing therapies for cancer treatment. Its leading program, TTI-621, is a SIRPaFc fusion protein designed to inhibit the CD47 signal, currently undergoing Phase I clinical trials for advanced relapsed or refractory hematologic malignancies, solid tumors, and mycosis fungoides. The company is also advancing TTI-622, another SIRPaFc protein in Phase I trials, and TTI-10001, a small molecule stimulator of interferon genes agonist in the discovery phase aimed at enhancing tumor immunity. Trillium Therapeutics, originally founded in 2004 and previously known as Stem Cell Therapeutics Corp., specializes in the discovery and development of protein therapeutics, including monoclonal antibodies and peptides, with a focus on cancer and other therapeutic areas.
Cleave Therapeutics, Inc. is a biopharmaceutical company based in San Francisco, California, founded in 2010. The company specializes in developing novel small-molecule drugs that target valosin-containing protein and other protein-degradation pathways critical for the survival of cancer cells. By focusing on these mechanisms, Cleave Therapeutics aims to create enzyme inhibitors that can effectively treat cancer and neurodegenerative diseases. Its research efforts are directed toward discovering innovative therapies that may improve patient outcomes in oncology.
Ardelyx, Inc. is a biopharmaceutical company focused on developing and commercializing innovative therapies for cardiorenal diseases. Headquartered in Fremont, California, Ardelyx's lead product candidate, tenapanor, has successfully completed Phase 3 clinical trials for treating irritable bowel syndrome with constipation and is also in Phase 3 trials for managing hyperphosphatemia in end-stage renal disease patients undergoing dialysis. The company is advancing RDX013, a small molecule potassium secretagogue, aimed at treating hyperkalemia. Ardelyx is also developing RDX5791, an oral NHE3 sodium transport inhibitor targeting constipation-predominant irritable bowel syndrome and excess dietary sodium absorption, currently in Phase 2 trials. Additionally, RDX002 and RDX009, which inhibit phosphate absorption and target type 2 diabetes, respectively, are in preclinical development. Ardelyx emphasizes non-systemic oral therapeutics that minimize side effects while effectively addressing metabolic disorders. Since its founding in 2007, the company has secured significant funding to support its research and development efforts.
Verona Pharma is a clinical-stage biopharmaceutical company headquartered in London, United Kingdom, that specializes in developing therapies for chronic respiratory diseases with significant unmet medical needs. The company's lead product candidate, ensifentrine, is an inhaled dual inhibitor of phosphodiesterase 3 and 4, functioning as both a bronchodilator and an anti-inflammatory agent. Ensifentrine is currently in Phase 2b clinical development as a nebulized formulation for the maintenance treatment of chronic obstructive pulmonary disease (COPD), and alternative delivery methods, including a dry powder inhaler and a pressurized metered-dose inhaler, are also being explored. In addition to COPD, Verona Pharma aims to develop ensifentrine for other respiratory conditions such as cystic fibrosis and asthma. Founded in 2005, the company is committed to improving the health and quality of life for individuals affected by these challenging diseases.
Mersana Therapeutics employs its biodegradable polymer platform to create new and better medicines. They are advancing their own clinical-stage pipeline of novel compounds with the potential to address multiple oncology indications.
It was founded in 2005 and headquartered in Cambridge, Massachusetts.
AVEO Oncology is a biopharmaceutical company engaged in the development and commercialization of targeted therapies for oncology and other unmet medical needs. The company's lead product, tivozanib, is an oral, once-daily inhibitor of vascular endothelial growth factor receptors, primarily indicated for renal cell carcinoma (RCC). AVEO has completed a Phase III trial (TIVO-3) for tivozanib and is conducting a Phase Ib/II trial combining it with Opdivo, an immune checkpoint inhibitor. Additionally, AVEO is developing Ficlatuzumab, an antibody currently in Phase II trials targeting various cancers, and AV-203, an anti-ErbB3 monoclonal antibody that has completed Phase I trials for esophageal cancer. The company's preclinical pipeline includes AV-380, aimed at cachexia, and AV-353, for pulmonary arterial hypertension. AVEO has established multiple collaboration agreements with various pharmaceutical companies and is involved in clinical trials assessing IMFINZI, an antibody targeting PD-L1. Headquartered in Boston, Massachusetts, AVEO was incorporated in 2001 and has a focus on advancing cancer therapeutics through its proprietary cancer biology platform.
Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing innovative therapeutics for patients with rare genetic disorders of hemoglobin, particularly sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing IMR-687, a once-daily oral therapy designed to be a potent small molecule inhibitor of PDE9. This therapeutic aims to address the significant health challenges associated with these conditions, which can lead to reduced healthy red blood cells and various complications. Imara was established following a collaboration between Cydan Development and H. Lundbeck A/S, with initial funding from notable life science investors.
TESARO, Inc. is an oncology-focused biopharmaceutical company established in 2010 and based in Waltham, Massachusetts. The company specializes in the identification, acquisition, development, and commercialization of cancer therapeutics and supportive care products. Its notable offerings include ZEJULA, an oral poly polymerase inhibitor used for the maintenance treatment of recurrent ovarian cancer, and VARUBI, a neurokinin-1 receptor antagonist designed to prevent chemotherapy-induced nausea and vomiting. TESARO is also advancing a range of immunotherapy candidates, such as TSR-042, TSR-022, and TSR-033, all currently in phase I clinical trials. The company has formed various collaboration and licensing agreements with industry partners, including AnaptysBio, Janssen Biotech, and Genentech. In January 2019, TESARO became a subsidiary of GlaxoSmithKline plc, further enhancing its capabilities in cancer treatment innovation.
Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is an oral therapy currently in Phase I/IIa clinical trials for the treatment of phenylketonuria. Additionally, Synlogic is developing SYNB1891, an intratumoral synthetic biotic medicine that is undergoing Phase I clinical trials for the treatment of solid tumors and lymphoma. The company is also exploring synthetic biotics for more common conditions, including inflammatory bowel disease and secondary hyperoxaluria. Synlogic has established a collaboration with Ginkgo Bioworks to advance its development of these innovative therapies.
Tarveda Therapeutics is focused on developing innovative cancer therapies specifically for solid tumors. The company specializes in miniature drug conjugates, with its lead candidate, PEN-866, targeting activated Heat Shock Protein 90 (HSP90) to deliver a potent topoisomerase 1 inhibitor, SN38, directly to tumor cells. Additionally, Tarveda is advancing another drug conjugate aimed at treating patients with neuroendocrine tumors and small cell lung cancer that express somatostatin receptor 2 (SSTR2). By leveraging its targeted drug delivery platform, Tarveda Therapeutics aims to improve treatment outcomes for patients with difficult-to-treat cancers while reducing potential side effects. Founded in 2011 and based in Watertown, Massachusetts, the company was previously known as Blend Therapeutics until its rebranding in 2016.
Atterocor, Inc. focuses on research and development of therapeutics for adrenal disease. Atterocor, Inc. was incorporated in 2012 and is based in Ann Arbor, Michigan.
Cascadian Therapeutics, a clinical-stage biopharmaceutical company, researches and develops, and sells therapeutic products for the treatment of cancer in the United States.
The company lead clinical-stage product candidate is tucatinib, an orally active and HER2-selective small molecule tyrosine kinase inhibitor, which is in two Phase Ib trials, one in combination with Kadcyla and another in combination with Xeloda and/or Herceptin. It is also developing Checkpoint kinase 1, a protein kinase that is in pre-clinical studies to regulate the cell division cycle, as well as to DNA damage and replication stress.
In addition, the company focuses on the development of novel antibodies and T-cell immunoreceptor with Ig and ITM domains, an inhibitory receptor expressed on T-cells and NK cells that negatively regulate immune response to cancers. It has a license agreement with Array BioPharma Inc. to develop, manufacture and commercialize tucatinib; research collaboration agreement with Sentinel Oncology Ltd. for the discovery of novel Chk1 inhibitors; and collaboration agreement with Adimab LLC for the discovery of novel antibodies against d immunotherapy targets in oncology.
Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing innovative therapeutics for patients with rare genetic disorders of hemoglobin, particularly sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing IMR-687, a once-daily oral therapy designed to be a potent small molecule inhibitor of PDE9. This therapeutic aims to address the significant health challenges associated with these conditions, which can lead to reduced healthy red blood cells and various complications. Imara was established following a collaboration between Cydan Development and H. Lundbeck A/S, with initial funding from notable life science investors.
AVEO Oncology is a biopharmaceutical company engaged in the development and commercialization of targeted therapies for oncology and other unmet medical needs. The company's lead product, tivozanib, is an oral, once-daily inhibitor of vascular endothelial growth factor receptors, primarily indicated for renal cell carcinoma (RCC). AVEO has completed a Phase III trial (TIVO-3) for tivozanib and is conducting a Phase Ib/II trial combining it with Opdivo, an immune checkpoint inhibitor. Additionally, AVEO is developing Ficlatuzumab, an antibody currently in Phase II trials targeting various cancers, and AV-203, an anti-ErbB3 monoclonal antibody that has completed Phase I trials for esophageal cancer. The company's preclinical pipeline includes AV-380, aimed at cachexia, and AV-353, for pulmonary arterial hypertension. AVEO has established multiple collaboration agreements with various pharmaceutical companies and is involved in clinical trials assessing IMFINZI, an antibody targeting PD-L1. Headquartered in Boston, Massachusetts, AVEO was incorporated in 2001 and has a focus on advancing cancer therapeutics through its proprietary cancer biology platform.
ObsEva SA is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, dedicated to developing innovative therapeutics for women's reproductive health and pregnancy. Founded in 2012 by Ernest Loumaye and André Chollet, the company focuses on addressing serious conditions such as endometriosis, heavy menstrual bleeding, and preterm labor. Its key products include Linzagolix, an oral gonadotropin-releasing hormone receptor antagonist aimed at treating pain from endometriosis and uterine fibroids, and OBE022, a selective prostaglandin F2α receptor antagonist designed for once-daily treatment of preterm labor. Additionally, ObsEva is advancing Nolasiban, an oral oxytocin receptor antagonist intended to enhance clinical pregnancy and live birth rates in women undergoing in vitro fertilization. The company operates primarily in Switzerland and is committed to improving outcomes for women facing reproductive health challenges.
Amplyx Pharmaceuticals is a small molecule drug development company focused on creating innovative therapies for life-threatening infections, particularly in patients with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in developing oral and injectable anti-infective drugs, including antifungal agents aimed at treating invasive fungal infections caused by pathogens such as Candida and Aspergillus. Amplyx employs a unique platform approach that enhances the efficacy and reduces the toxicity of existing drugs by combining them with additional small molecules. This strategy addresses critical treatment needs in hospital settings and provides effective therapy options for patients after discharge, particularly those undergoing chemotherapy and other immunosuppressive treatments.
Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. Therachon is headquartered in Basel, Switzerland, with research labs in Nice, France.
Akari Therapeutics is a clinical-stage biopharmaceutical company based in London, focused on developing and commercializing innovative treatments for rare and orphan autoimmune and inflammatory diseases. The company's lead product candidate, Coversin, is a second-generation complement inhibitor currently undergoing Phase II clinical trials for conditions such as paroxysmal nocturnal hemoglobinuria, Guillain-Barré syndrome, and atypical hemolytic uremic syndrome. Akari Therapeutics aims to provide life-transforming therapies that target specific pathways, including the complement component 5 (C5) and leukotriene B4 (LTB4) pathways. Founded in 2015, the company is dedicated to addressing unmet medical needs in the field of autoimmune and inflammatory diseases.
Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.
Ra Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapeutics for diseases linked to the complement system's excessive activation. Utilizing a proprietary peptide chemistry platform, the company creates synthetic macrocyclic peptides that possess the specificity of antibodies and the advantageous properties of small molecules. Its primary product candidate, Zilucoplan, is an injectable treatment that has completed Phase II clinical trials for generalized myasthenia gravis and paroxysmal nocturnal hemoglobinuria, as well as a Phase Ib trial for patients with renal impairment. Ra Pharmaceuticals is also exploring pre-clinical programs targeting various complement factors for conditions such as C3 glomerulonephritis and autoimmune diseases. Additionally, the company has a collaboration with Merck & Co. to identify orally available cyclic peptides for cardiovascular targets. Founded in 2008, Ra Pharmaceuticals operates as a subsidiary of UCB SA.
Clementia Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Montreal, Canada, focused on developing innovative treatments for patients with ultra-rare bone disorders and other diseases characterized by high unmet medical needs. Founded in 2010, the company specializes in disease-modifying therapies, with its lead product candidate being palovarotene, an oral small molecule. Palovarotene has demonstrated significant potential in preventing abnormal bone formation and fibrosis across various tissues. The candidate is currently undergoing evaluation in the Phase III MOVE trial, targeting conditions such as fibrodysplasia ossificans progressive and multiple osteochondromas, as well as dry eye disease. Clementia's commitment to addressing debilitating health issues highlights its role in advancing treatment options for affected patients.
Ardelyx, Inc. is a biopharmaceutical company focused on developing and commercializing innovative therapies for cardiorenal diseases. Headquartered in Fremont, California, Ardelyx's lead product candidate, tenapanor, has successfully completed Phase 3 clinical trials for treating irritable bowel syndrome with constipation and is also in Phase 3 trials for managing hyperphosphatemia in end-stage renal disease patients undergoing dialysis. The company is advancing RDX013, a small molecule potassium secretagogue, aimed at treating hyperkalemia. Ardelyx is also developing RDX5791, an oral NHE3 sodium transport inhibitor targeting constipation-predominant irritable bowel syndrome and excess dietary sodium absorption, currently in Phase 2 trials. Additionally, RDX002 and RDX009, which inhibit phosphate absorption and target type 2 diabetes, respectively, are in preclinical development. Ardelyx emphasizes non-systemic oral therapeutics that minimize side effects while effectively addressing metabolic disorders. Since its founding in 2007, the company has secured significant funding to support its research and development efforts.
XTuit Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing therapeutics that target the disease-promoting microenvironment associated with fibrotic diseases and cancer. Founded in 2011 and based in Waltham, Massachusetts, the company works on innovative pharmaceutical preparations aimed at diagnosing and treating oncological and inflammatory conditions. Its drug development pipeline includes micro-environment activated therapeutics that utilize pleiotropic mechanisms to inhibit the synthesis and stabilization of extracellular matrix and to silence activated stromal cells. Through these novel approaches, XTuit Pharmaceuticals seeks to improve cancer therapies by addressing the tumor microenvironment effectively.
CRISPR Therapeutics AG is a gene editing company dedicated to developing transformative gene-based medicines for serious human diseases using its proprietary CRISPR/Cas9 technology. This revolutionary platform allows for precise modifications to genomic DNA, enabling the company to target a variety of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The lead product candidate, CTX001, is an ex vivo CRISPR gene-edited therapy aimed at treating transfusion-dependent beta thalassemia and severe sickle cell disease by engineering patients' hematopoietic stem cells to produce elevated levels of fetal hemoglobin. Additionally, CRISPR Therapeutics is advancing other therapies, including CTX110, an allogeneic CAR-T therapy for certain malignancies, and additional programs targeting conditions such as multiple myeloma, solid tumors, diabetes, and various genetic disorders. The company collaborates with strategic partners such as Bayer Healthcare, Vertex Pharmaceuticals, and ViaCyte, and is headquartered in Zug, Switzerland.
CRISPR Therapeutics AG is a gene editing company dedicated to developing transformative gene-based medicines for serious human diseases using its proprietary CRISPR/Cas9 technology. This revolutionary platform allows for precise modifications to genomic DNA, enabling the company to target a variety of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The lead product candidate, CTX001, is an ex vivo CRISPR gene-edited therapy aimed at treating transfusion-dependent beta thalassemia and severe sickle cell disease by engineering patients' hematopoietic stem cells to produce elevated levels of fetal hemoglobin. Additionally, CRISPR Therapeutics is advancing other therapies, including CTX110, an allogeneic CAR-T therapy for certain malignancies, and additional programs targeting conditions such as multiple myeloma, solid tumors, diabetes, and various genetic disorders. The company collaborates with strategic partners such as Bayer Healthcare, Vertex Pharmaceuticals, and ViaCyte, and is headquartered in Zug, Switzerland.
PhaseBio Pharmaceuticals is a clinical-stage biopharmaceutical company based in Malvern, Pennsylvania, focused on developing innovative therapies for cardiopulmonary diseases. Its lead product candidate, PB2452, is a reversal agent for the antiplatelet drug ticagrelor and is currently undergoing Phase III clinical trials for patients experiencing uncontrolled bleeding or requiring urgent surgery. The company is also advancing PB1046, a fusion protein aimed at treating pulmonary arterial hypertension, which is in Phase IIb clinical trials. Additionally, PhaseBio is developing PB6440, an oral agent intended for the treatment of resistant hypertension. Founded in 2002, the company leverages proprietary recombinant biopolymers to enhance the stability and efficacy of its drug candidates.
Mersana Therapeutics employs its biodegradable polymer platform to create new and better medicines. They are advancing their own clinical-stage pipeline of novel compounds with the potential to address multiple oncology indications.
It was founded in 2005 and headquartered in Cambridge, Massachusetts.
Strongbridge Biopharma’s primary focus has been to build its franchise around rare endocrine disorders, which includes product candidates for the treatment of endogenous Cushing’s syndrome and acromegaly, two rare diseases with a high unmet need for innovative treatment options. Given the well-identified and concentrated prescriber base addressing its target markets, the Company intends to use a small, focused sales force to market its products, if approved, in the United States, European Union and other key global markets. The Company intends to identify and in-license or acquire products or product candidates that would be complementary to its existing rare endocrine franchise or that would form the basis for new rare disease franchises.
Tarveda Therapeutics is focused on developing innovative cancer therapies specifically for solid tumors. The company specializes in miniature drug conjugates, with its lead candidate, PEN-866, targeting activated Heat Shock Protein 90 (HSP90) to deliver a potent topoisomerase 1 inhibitor, SN38, directly to tumor cells. Additionally, Tarveda is advancing another drug conjugate aimed at treating patients with neuroendocrine tumors and small cell lung cancer that express somatostatin receptor 2 (SSTR2). By leveraging its targeted drug delivery platform, Tarveda Therapeutics aims to improve treatment outcomes for patients with difficult-to-treat cancers while reducing potential side effects. Founded in 2011 and based in Watertown, Massachusetts, the company was previously known as Blend Therapeutics until its rebranding in 2016.
GSMS is a pharmaceutical company that specializes in labeling and serialization services, as well as repackaging and distribution. The company caters primarily to the Federal government, supplying generic therapeutic treatments to various entities, including mail-order pharmacies, federal and state pharmacies, and local government programs. GSMS focuses on meeting the unique labeling and dispensing requirements of its clients while ensuring that its products are offered at an affordable price. Through its comprehensive services, the company aims to enhance the accessibility of essential therapeutic treatments across multiple sectors.
Sagimet Biosciences is a biotechnology company that focuses on developing novel therapeutics to treat important diseases such as liver disease NASH and specific cancers, with a focus on targeting dysfunctional metabolic pathways. It was founded in 2007 and headquartered in San Mateo, California.
Ziarco, Inc. develops therapeutic agents for treating inflammatory and allergic diseases. The company was founded in 2012 and is based in Palo Alto, California.
Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is an oral therapy currently in Phase I/IIa clinical trials for the treatment of phenylketonuria. Additionally, Synlogic is developing SYNB1891, an intratumoral synthetic biotic medicine that is undergoing Phase I clinical trials for the treatment of solid tumors and lymphoma. The company is also exploring synthetic biotics for more common conditions, including inflammatory bowel disease and secondary hyperoxaluria. Synlogic has established a collaboration with Ginkgo Bioworks to advance its development of these innovative therapies.
TRACON Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing and commercializing targeted therapeutics for cancer and wet age-related macular degeneration (AMD). The company's lead product is envafolimab, an investigational PD-L1 single-domain antibody aimed at treating soft tissue sarcoma. Other notable candidates include DE-122, which is undergoing a randomized Phase IIa study for wet AMD; TRC102, a small molecule currently in multiple clinical trials for various cancers, including mesothelioma and lung cancer; TRC253, in Phase II trials for metastatic castration-resistant prostate cancer; and TJ004309, a CD73 antibody in Phase I development for solid tumors. TRACON has established collaborations and licensing agreements with several organizations, including 3D Medicines and Janssen Pharmaceutica, to advance its product development. TRACON was originally founded in 2004 and underwent a name change from Lexington Pharmaceuticals in 2005.
Dermira is a specialty biopharmaceutical company focused on bringing innovative and differentiated products to dermatologists and their patients. Dermira’s portfolio of five product candidates targets significant market opportunities.
It was founded in 2010 and headquartered in Menlo Park, California.
Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is an oral therapy currently in Phase I/IIa clinical trials for the treatment of phenylketonuria. Additionally, Synlogic is developing SYNB1891, an intratumoral synthetic biotic medicine that is undergoing Phase I clinical trials for the treatment of solid tumors and lymphoma. The company is also exploring synthetic biotics for more common conditions, including inflammatory bowel disease and secondary hyperoxaluria. Synlogic has established a collaboration with Ginkgo Bioworks to advance its development of these innovative therapies.
Cerecor Inc. is a biopharmaceutical company dedicated to the development and commercialization of treatments for rare pediatric and orphan diseases, with a particular focus on inherited metabolic disorders. The company's pipeline includes therapies such as CERC-801, CERC-802, and CERC-803, which have completed phase I clinical trials. Additionally, Cerecor is developing CERC-007, an anti-IL-18 monoclonal antibody for autoimmune inflammatory diseases, and CERC-006, a dual mTOR inhibitor for complex lymphatic malformations. Other notable developments include CERC-002 for pediatric-onset Crohn's disease, CERC-913 for mitochondrial disorders, and CERC-005 for rare auto-inflammatory diseases. Cerecor also offers Millipred, an oral prednisolone for various inflammatory conditions. Founded in 2011 and headquartered in Rockville, Maryland, the company was previously known as Ceregen Corporation before rebranding in March 2011.
Loxo Oncology, Inc., a biopharmaceutical company, develops and sells medicines for patients with genetically defined cancers in the United States. Its lead product candidate comprises larotrectinib, an oral selective inhibitor of tropomyosin receptor kinase (TRK), which is in adult Phase 1 trial, a pediatric Phase 1/2 trial, and an adult/adolescent Phase 2 trial for the treatment of patients with tumor types, such as lung, head and neck, melanoma, colorectal, sarcoma, and breast cancer. The company’s preclinical programs include LOXO-195, a drug candidate in preclinical development to address predicted acquired resistance mechanisms; RET inhibitor that optimizes potency for rearranged during transfection (RET) fusion proteins, mutations, and anticipated mechanisms of acquired resistance; and FGFR inhibitor that enables potently inhibiting FGFR isoforms comprising four isoforms with tyrosine kinase domains. It has a drug discovery collaboration agreement with Array BioPharma, Inc.; strategic partnership agreement with Illumina, Inc.; research collaboration agreement with Veracyte, Inc.; and strategic partnership agreement with Amoy Diagnostics Co., Ltd. and PREMIA Holdings (HK) Ltd. Loxo Oncology, Inc. was founded in 2013 and is headquartered in Stamford, Connecticut. Loxo Oncology, Inc. is a subsidiary of Eli Lilly and Company.
Mersana Therapeutics employs its biodegradable polymer platform to create new and better medicines. They are advancing their own clinical-stage pipeline of novel compounds with the potential to address multiple oncology indications.
It was founded in 2005 and headquartered in Cambridge, Massachusetts.
Lumena Pharmaceuticals focuses on developing oral therapeutics for rare liver diseases, aiming to enhance liver function and alleviate disease symptoms. The company's clinical-stage product candidates are designed to selectively target a transporter in the intestine, minimizing systemic absorption and reducing the risk of toxicities. Lumena's lead candidate, LUM001, has been thoroughly evaluated in over 12 clinical studies involving more than 1,400 subjects, positioning it for rapid advancement in clinical trials for both pediatric and adult patients with various forms of cholestatic liver disease. In addition to its primary objective of creating novel treatments for rare liver conditions, Lumena's innovative therapeutic approach also holds potential for addressing metabolic diseases affecting the liver, which represent a significant and growing health concern among both children and adults.
Aurinia Pharmaceuticals Inc. is a clinical stage biopharmaceutical company based in Victoria, British Columbia, that specializes in developing and commercializing therapies for serious diseases with significant unmet medical needs. The company is primarily focused on the investigational drug voclosporin, which is being developed for the treatment of lupus nephritis, focal segmental glomerulosclerosis, and dry eye syndrome. Aurinia aims to address the needs of targeted patient populations in both the United States and China through its innovative therapeutic approaches.
Lumos Pharma is a clinical-stage biopharmaceutical company based in Austin, Texas, dedicated to developing and commercializing therapeutics for severe, rare, and genetic diseases. Its lead candidate, LUM-201, is an oral growth hormone stimulating small molecule currently undergoing Phase 2 clinical trials for pediatric growth hormone deficiency. The company also holds a licensing agreement with Ellipses Pharma Limited to develop and commercialize nanoparticle formulations for oncology indications. Additionally, Lumos Pharma is focused on treating Creatine Transporter Deficiency, leveraging technology from the University of Cincinnati and collaborating with Key Opinion Leaders and the National Institutes of Health. Founded in 2011, Lumos Pharma aims to address unmet medical needs through innovative therapies.
Tarveda Therapeutics is focused on developing innovative cancer therapies specifically for solid tumors. The company specializes in miniature drug conjugates, with its lead candidate, PEN-866, targeting activated Heat Shock Protein 90 (HSP90) to deliver a potent topoisomerase 1 inhibitor, SN38, directly to tumor cells. Additionally, Tarveda is advancing another drug conjugate aimed at treating patients with neuroendocrine tumors and small cell lung cancer that express somatostatin receptor 2 (SSTR2). By leveraging its targeted drug delivery platform, Tarveda Therapeutics aims to improve treatment outcomes for patients with difficult-to-treat cancers while reducing potential side effects. Founded in 2011 and based in Watertown, Massachusetts, the company was previously known as Blend Therapeutics until its rebranding in 2016.
Zosano Pharma Corporation is a clinical stage biopharmaceutical company based in Fremont, California, established in 2006. The company specializes in developing therapeutics for migraine sufferers through its innovative intracutaneous microneedle delivery system. Its primary product candidate, Qtrypta (M207), is a proprietary formulation of zolmitriptan aimed at treating migraines. Zosano Pharma is committed to discovering and developing drug candidates that meet significant unmet medical needs and have strong commercialization potential.