Biotechnology Stocks Profiles
Biotechnology (biotech) stocks are publicly traded companies that develop pharmaceutical drugs used in the treatment of disease. You can use Barchart's Biotech Stocks list to find investment opportunities and compare stocks involved in this hot industry.
Ginkgo Bioworks Hldgs Inc (DNA)Full Profile
Ginkgo Bioworks platform is enabling biotechnology applications across diverse markets, from food and agriculture to industrial chemicals to pharmaceuticals. Ginkgo Bioworks, formerly known as Soaring Eagle Acquisition Corp., is based in NEW YORK.
Adaptive Biotechnologies Corp (ADPT)Full Profile
Adaptive Biotechnologies Corporation is a commercial-stage biotech company. It develops and commercializes immune-driven clinical products to transform the diagnosis and treatment of disease. The company's product pipeline includes immunoSEQ, clonoSEQ and cellular therapy which are in clinical stage. Adaptive Biotechnologies Corporation is based in Seattle, Washington.
Edgewise Therapeutics Inc (EWTX)Full Profile
Edgewise Therapeutics Inc. is a clinical-stage biopharmaceutical company. It is focused on developing orally bioavailable, small molecule therapies for rare muscle disorders. Edgewise Therapeutics Inc. is based in Boulder, United States.
Kiniksa Pharmaceuticals Ltd (KNSA)Full Profile
Kiniksa Pharmaceuticals Ltd. is a clinical-stage biopharmaceutical company. It focuses on discovering, acquiring, developing and commercializing therapeutic medicines for patients suffering from debilitating diseases with significant unmet medical need. Kiniksa Pharmaceuticals Ltd. is based in Massachusetts, United States.
Relay Therapeutics Inc (RLAY)Full Profile
Relay Therapeutics Inc. is a clinical-stage precision medicines company. It focuses on enhancing small molecule therapeutic discovery in targeted oncology. The company's product pipeline consist RLY-1971, RLY-4008 and RLY-PI3K1047 which are in clinical stage. Relay Therapeutics Inc. is based in Cambridge, Massachusetts.
Crispr Therapeutics Ag (CRSP)Full Profile
CRISPR Therapeutics AG is a leading gene editing company focused on developing CRISPR/Cas9-based therapeutics. The company is rapidly leveraging its CRISPR/Cas9 gene-editing platform to make therapies for the treatment of hemoglobinopathies, cancer, diabetes and other diseases.
Vir Biotechnology Inc (VIR)Full Profile
Vir Biotechnology Inc. is a clinical-stage immunology company. It is focused on combining immunologic insights to treat and prevent serious infectious diseases. The company's development pipeline consists of product candidates targeting the hepatitis B virus, influenza A, human immunodeficiency virus and tuberculosis. Vir Biotechnology Inc. is based in San Francisco, United States.
Acadia Pharmaceutica (ACAD)Full Profile
Acadia Pharmaceuticals Inc. is a biopharmaceutical company focused on developing innovative medicines to address the unmet medical needs in central nervous system (CNS) disorders. The company's sole marketed drug Nuplazid (pimavanserin) is the first and the only FDA-approved treatment for hallucinations and delusions associated with Parkinson's disease psychosis. The company suffered a setback when the FDA issued a complete response letter to the supplemental new drug application for Nuplazid for treating dementia-related psychosis. Acadia resubmitted the supplemental new drug application to the FDA for pimavanserin for the treatment of hallucinations and delusions associated with Alzheimer's disease psychosis. Notably, several additional studies on Nuplazid n targeting different CNS indications, such as'dementia-related psychosis, schizophrenia inadequate response, schizophrenia negative symptoms and as an adjunctive treatment of major depressive disorder (MDD) are currently underway.
Aadi Biosciences Inc (AADI)Full Profile
Aadi Bioscience Inc. is a clinical-stage biopharmaceutical company. It focuses on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes. Aadi Bioscience Inc., formerly known as Aerpio Pharmaceuticals Inc., is based in LOS ANGELES.
Novavax Inc (NVAX)Full Profile
Novavax, Inc. is a biotechnology company engaged in developing innovative vaccines to prevent serious infectious diseases. The company is also developing proprietary immune stimulating saponin-based adjuvants, namely Matrix-M, which already enhanced immune responses and were well-tolerated in multiple studies.'Novavax developed its own coronavirus vaccine NVX-CoV2373. The company is currently marketing two versions of NVX-CoV2373, one marketed in partnership with the Serum Institute of India under the trade name Covovax and another version produced by Novavax and marketed under the trade name Nuvaxovid.'Novavax developed NanoFlu, its recombinant quadrivalent seasonal influenza vaccine candidate with Matrix M adjuvant for senior patients aged 65 years and above.'ResVax a respiratory syncytial virus vaccine is being developed to protect infants by immunizing mothers against the disease. Novavax is discussing the opportunity to bring ResVax to the market globally with the help of commercial partners.
Seagen Inc (SGEN)Full Profile
Seagen, Inc., a biotechnology company, focuses on developing & commercializing therapies targeted for the treatment of cancer. Adcetris, a lead FDA approved marketed product at Seagen, is used for treating Hodgkin lymphoma, certain T-cell lymphomas, other cancers in the U.S., Europe & several countries. Seagen has an agreement with Takeda Pharmaceutical Company Ltd. for the further development and commercialization of Adcetris. Seagen retains all rights to sell Adcetris in the U.S. and Canada, while Takeda has commercial rights to the drug in the rest of the world. The FDA granted approvals to Seagen's several drugs named Padcev, for treating advanced/metastatic urothelial cancer; Tukysa and Tivdak for treating adult patients with locally advanced/metastatic HER2-positive breast cancer and for the treatment of recurrent/metastatic cervical cancer in adult patients respectively. The company also has license agreements with partners like Merck, Roche, GlaxoSmithKline, Astellas, AbbVie, Bristol-Myers and Genmab.
Moderna Inc (MRNA)Full Profile
Moderna, Inc. is a clinical-stage pharmaceutical company, primarily focused on discovering and developing messenger RNA (mRNA) based therapies. The company's mRNA based COVID-19 vaccine, Spikevax (mRNA-1273), is approved for use in adults in the United States. The vaccine is also authorized for emergency use in several countries as COVID-19 vaccine. The company has also developed booster doses of its COVID-19 vaccine and is developing variant-specific booster candidates.
Rogers Corp (ROG)Full Profile
Rogers Corporation is a global leader in engineered materials to power, protect, and connect their world. With more than 180 years of materials science experience, Rogers delivers high-performance solutions that enable clean energy, internet connectivity, and safety and protection applications, as well as other technologies where reliability is critical. Rogers delivers Power Electronics Solutions for energy-efficient motor drives, e-Mobility and renewable energy; Elastomeric Material Solutions for sealing, vibration management and impact protection in mobile devices, transportation interiors, industrial equipment and performance apparel; and Advanced Connectivity Solutions for wireless infrastructure, automotive safety and radar systems. Headquartered in Arizona (USA), Rogers operates manufacturing facilities in the United States, China, Germany, Belgium, Hungary, and South Korea, with joint ventures and sales offices worldwide.
Ascendis Pharma ADR (ASND)Full Profile
Ascendis Pharma A/S is a biopharmaceutical company. The company's product pipeline consist of TransCon Human Growth Hormone, TransCon Treprostinil, TransCon Insulin, TransCon Peptide and TransCon Ranibizumab, which are in different clinical stage, for the treatments of hormone deficiency, endocrinology, central nervous system disorders, infectious diseases and diabetes. Its technology includes TransCon which develop prodrug therapies. Ascendis Pharma A/S is based in Denmark.
Biontech Se ADR (BNTX)Full Profile
BioNTech SE is a biotechnology company. It develops and commercializes immunotherapies for cancer and other diseases. BioNTech SE is based in Mainz, Germany.
Vanda Pharmaceuticals (VNDA)Full Profile
Vanda Pharmaceuticals Inc. is a biopharmaceutical company focused on the development and commercialization of clinical-stage product candidates for central nervous system disorders. It is developing important new medicines to improve the lives of patients. It uses new technologies, including genetics & genomics, to inform our drug discovery, our clinical trials, and our commercial positioning of our compounds. The Company has three product candidates in clinical development. It's lead product candidate, iloperidone, is a compound for the treatment of schizophrenia & bipolar disorder & is in a Phase III clinical trial for schizophrenia. It's second product candidate, is a compound for the treatment of insomnia & depression which is currently in a Phase III clinical trial for insomnia. It's third product candidate, is a compound for the treatment of excessive sleepiness & is ready for a Phase II clinical trial.
Incyte Corp (INCY)Full Profile
Incyte Corporation is a biopharmaceutical company focused on the discovery, development and commercialization of proprietary therapeutics. The company conducts its European clinical development operations in Geneva, Switzerland. Incyte's lead drug, Jakafi (ruxolitinib), is a first-in-class JAK1/JAK2 inhibitor, approved in the United States for the treatment of patients with polycythemia vera ('PV'),who have had an inadequate response to or are intolerant to hydroxyurea. It is also approved for the treatment of patients with intermediate or high-risk myelofibrosis (MF), including primary MF, post-PV MF, and post-essential thrombocythemia MF. The drug is also approved in the United States for treatment of steroid-refractory acute graft-versus-host disease (GVHD) in adult and pediatric patients aged 12 years or older.
Gilead Sciences Inc (GILD)Full Profile
Gilead Sciences is a pioneer in developing drugs for the treatment of human immunodeficiency virus, liver diseases, hematology/oncology diseases and inflammation/respiratory diseases. The company has a strong HIV franchise with key HIV/AIDS therapies like tenofovir alafenamide based products - Genvoya, Odefsey, Descovy, Biktarvy and Truvada. The portfolio also includes hepatitis C virus drugs like Harvoni and Epclusa and HBV drug. The first cell therapy approved for the treatment of adult patients with relapsed or refractory large B-cell lymphoma, has diversified Gilead's portfolio. Tecartus, another CAR T-cell therapy, was granted an accelerated approval in the United States for the treatment of relapsed or refractory mantle cell lymphoma. The company is also working on diversifying and growing its business beyond antivirals into other therapeutic areas. Gilead is also making inroads in the oncology space with strategic collaborations and acquisitions.
Johnson & Johnson (JNJ)Full Profile
Johnson & Johnson's biggest strength is its diversified business model. It operates through pharmaceuticals, medical devices and consumer products divisions. Its diversification helps it to withstand economic cycles more effectively. J&J has one of the largest research and development (R&D) budget among pharma companies. J&J's worldwide business is divided into three segments: Pharmaceutical, Medical Devices and Consumer. The company has several drugs covering a broad range of areas such as neuroscience, cardiovascular & metabolism, immunology, oncology, pulmonary hypertension and infectious diseases and vaccines. The Medical Devices Segment offers products in the orthopedics, surgery, interventional solutions and vision markets. The Consumer Segment segment includes a broad range of products covering the areas of baby care, beauty/skin health, oral care, wound care and womens' health care, as well as over-the-counter (OTC) pharmaceutical products.
Amgen Inc (AMGN)Full Profile
Amgen Inc. is the leading biotech companies world wide, with a strong presence in the oncology/hematology, cardiovascular disease, neuroscience, inflammation, bone health & nephrology and neuroscience markets. It developed two most successful drugs, Epogen & Neupogen. Amgen successfully launched two next-generation products, Aranesp & Neulasta and also a biosimilar drug, Prolia/Xgeva. The acquisition of Immunex Corp gave Amgen access to the multi-blockbuster drug, Enbrel. Other products are Repatha, Blincyto, Parsabiv, Evenity, Aimovig, Lumakras/Lumykras, Kanjinti, Mvasi & Amgevita biosimilars. Amgen also has a promising pipeline of cancer drugs. It has one of the strongest cash positions in the biotech sector, which could be used to acquire more pipeline assets that could fuel long-term growth. The erythropoiesis-stimulating agents and granulocyte colony-stimulating factor franchise comprise Epogen/Aranesp and Neupogen/Neulasta respectively. Biosimilar drugs are also a key part of Amgen's growth strategy.
Novartis Ag ADR (NVS)Full Profile
Novartis has one of the strongest and broadest portfolio of oncology drugs and generics. It continues to build depth in 5 core therapeutic areas Cardio-Renal, Immunology, Neuroscience, Oncology and Hematology, strength in technology platforms - Targeted Protein Degradation, Cell Therapy, Gene Therapy, Radioligand Therapy, and xRNA. Novartis efforts to strengthen its wide and deep oncology portfolio by developing breakthrough treatments. Cosentyx, Entresto, Kesimpta, Zolgensma, Kisqali and Leqvio. In March 2015, Novartis acquired certain oncology products and pipeline compounds from Glaxo. In exchange, it sold its non-influenza Vaccines business to Glaxo. Also, the company has spun off the Alcon business into a separate company. In January 2015, Novartis divested its Animal Health division to Lilly. In July 2015, the company divested its influenza vaccines business to CSL Limited. Novartis has also acquired The Medicines Company, adding Leqvio, to its portfolio. Novartis sold its investment in Roche.
Ligand Pharm (LGND)Full Profile
Ligand, being a biotechnology company, focuses on developing/acquiring royalty revenue generating assets and coupling them with a lean corporate cost structure. Ligand's Captisol formulation technology has allowed it to enter into several licensing deals and generate royalties. Captisol is a well validated chemically modified cyclodextrin that is designed to improve safety and solubility, stability, and bioavailability or lessen the volatility, irritation, smell or taste of drugs. Ligand has merged with CyDex. By this, it added an antibody-generating platform, OmniAb, to the company's portfolio. Its other technology platforms include antigen discovery platform & protein expression platform. All these technologies have created a strong platform for Ligand to seek new licenses and partnerships. Ligand has partnership agreements with leading healthcare companies like Novartis, Amgen, Merck, Pfizer, Celgene, Gilead & Lilly, etc. The company continues to buy smaller companies to increase its technology platforms.
Astrazeneca Plc ADR (AZN)Full Profile
AstraZeneca plc, is one of the largest biopharmaceutical companies in the world. AstraZeneca's business can be broken down into separate lines based on therapeutic classes. These include cardiovascular, respiratory, immunology, oncology, rare diseases and other.In July 2021 AstraZeneca closed the acquisition of rare disease drugmaker, Alexion. The acquisition added Alexion's five marketed rare disease products and its pipeline of immune-mediated rare disease candidates.In order to bolster its respiratory portfolio, AstraZeneca acquired rights to Allergan's branded respiratory business in the U.S. and Canada in Mar 2015. The company also acquired Takeda's core respiratory business in May 2016.In June 2007, AstraZeneca acquired biotechnology company MedImmune. In Aug 2011, AstraZeneca sold its Astra Tech business to DENTSPLY. AstraZeneca entered into a number of deals and struck agreements with companies such as FibroGen and Daiichi Sankyo.
Bristol-Myers Squibb Company (BMY)Full Profile
Bristol-Myers Squibb is a one of the leading global specialty biopharmaceutical companies focused on the development of treatments targeting serious diseases like cancer, inflammatory, immunologic, cardiovascular or fibrotic diseases. Backed by its blockbuster immuno-oncology drug, Opdivo, Bristol-Myers has a strong oncology portfolio, comprising other drugs like Revlimid, Pomalyst, Sprycel, Yervoy and Empliciti. It also has important immunology and cardiovascular drugs like Orencia and Eliquis. It's experiencing growth in both the Eliquis brand and the market, while also advancing its Factor XIa inhibitor program. After the sale of the global Diabetes business to AstraZeneca and the discontinuation of discovery research efforts in virology, it's focusing solely on research in core therapeutic areas like oncology, immuno-oncology, immunoscience, cardiovascular, fibrosis and genetically defined diseases. It acquried Celgene Corporation and MyoKardia. It received regulatory approvals for Reblozyl and Inrebic.
Biogen Inc (BIIB)Full Profile
Biogen Inc. is one of the world's leading biotechnology companies which focuses on developing innovative therapies for treating serious neurological and neurodegenerative diseases, including its core growth areas of multiple sclerosis (MS) and neuroimmunology, Alzheimer's disease (AD) and dementia, movement disorders including Parkinson's disease, neuromuscular disorders, including spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS) and ophthalmology. Key multiple sclerosis/MS drugs in its portfolio are Tecfidera, Vumerity, Avonex, Plegridy, Fampyra and Tysabri. Other approved/marketed products include Fumaderm (severe psoriasis), Spinraza (spinal muscular atrophy (SMA)) and Aduhelm (Alzheimer's disease).
Vertex Pharmaceutic (VRTX)Full Profile
Vertex Pharmaceuticals Incorporated is focused on the discovery, development, and commercialization of small molecule drugs targeting serious diseases. The company's main area of focus is cystic fibrosis. The company's lead marketed products are Trikafta, Symdeko/Symkevi, Orkambi and Kalydeco, which are collectively approved to treat the people with CF in North America, Europe and Australia. Trikafta, Vertex's triple combination regimen, was approved by the FDA for the treatment of CF in people aged 12 years and older who have at least one F508del mutation. While CF remains the main area of focus, Vertex is also developing treatments for sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, type 1 diabetes, pain, alpha-1 antitrypsin deficiency and muscular dystrophies. Pimodivir/VX-787, for the treatment of influenza, was out-licensed to Janssen while oncology candidates VX-970, VX-984 and VX-803 were divested to Merck KGaA.
Biomarin Pharmaceuticals (BMRN)Full Profile
BioMarin Pharmaceutical Inc. focuses on the development and commercialization of treatments for serious life-threatening medical conditions, mostly for children. The company's portfolio comprises seven marketed products namely, Aldurazyme, Naglazyme, Kuvan a rare genetic enzyme deficiency disorder), Vimizim , Brineura, Palynziq and Voxzogo. BioMarin has a collaboration agreement with Sanofi's subsidiary Genzyme for Aldurazyme. Genzyme is BioMarin's sole customer for Aldurazyme and is responsible for marketing and selling Aldurazyme to third parties.'The newest drug in BioMarin's portfolio, Voxzogo for treating achondroplasia, the most common form of dwarfism. BioMarin's biologics license application (BLA) for Roctavian/valoctocogene roxaparvovec (valrox), a gene therapy for severe hemophilia A, was given a complete response letter (CRL) by the FDA.
Merck & Company (MRK)Full Profile
Merck & Co. boasts more than six blockbuster drugs in its portfolio with PD-L1 inhibitor, Keytruda, approved for several types of cancer. Keytruda has played an instrumental role in driving Merck's steady revenue growth in the past few years. Well-known products in Merck's portfolio include Keytruda, Simponi , Januvia and Janumet, Bridion, Isentress, ProQuad, Gardasil, Pneumovax 23, RotaTeq and Belsomra. Merck made its biggest acquisition of Schering-Plough and sold off its Consumer Care business to Bayer. Other key acquisitions include Idenix Pharmaceuticals, Cubist Pharmaceuticals, Rigontec, ArQule and Acceleron Pharma. IMerck spun off products from its Women's Health unit, legacy drugs and biosimilar products into a new publicly traded company called Organon & Co.
Gsk Plc ADR (GSK)Full Profile
GSK plc is a biopharma company. Its vaccine portfolio protects people from meningitis, shingles, flu, polio, measles and many more. GSK plc, formerly known as GLAXOSMITHKLINE, is based in Brentford, the United Kingdom.
Illumina Inc (ILMN)Full Profile
Illumina Inc. is a life sciences company, which provides tools and integrated systems for analysis of genetic variation and function. Using its proprietary technologies, the company provides innovative sequencing and array-based solutions for genotyping, copy number variation analysis, methylation studies, and gene expression profiling of DNA and RNA. Its customers include leading genomic research centers, academic institutions, government laboratories, hospitals and reference laboratories as well as pharmaceutical, biotechnology, agrigenomics, commercial molecular diagnostic and consumer genomics companies. Illumina generates revenue from two segments - Product and Service. Product are primarily attributed to the partnerships and collaborations to develop distributable clinical in-vitro diagnostics for Illumina sequencers. Service include genotyping and sequencing services as well as instrument maintenance contracts.
Regeneron Pharmaceuticals (REGN)Full Profile
Regeneron is a biotechnology company focused on the discovery, development and commercialization of treatments targeting serious medical conditions. The company's portfolio boasts nine marketed drugs - Eylea, Dupixent, Praluent, Kevzara, Libtayo, Evkeeza, Inmazeb Arcalyst and Zaltrap. The company also developed an antibody cocktail for COVID-19, REGEN-COV. REGEN-COV is a cocktail of two monoclonal antibodies and was designed specifically to block the infectivity of SARS-CoV-2, the virus that causes COVID-19. Regeneron has a collaboration agreement with Roche for the same. While Regeneron has co-developed Eylea with Bayer's HealthCare unit, Praluent was co-developed with Sanofi. Regeneron collaborated with Bayer for the joint development and commercialization of co-formulated combinations of Eylea, rinucumab and nesvacumab for the treatment of ocular diseases or disorders outside the United States.
Sanofi-Aventis S.A. ADR (SNY)Full Profile
Sanofi-Aventis manufactures & markets prescription drugs across countries. It focuses on major therapeutic areas such as multiple sclerosis, cardiovascular, immunology, neurology, oncology, rare blood disorders & diabetes, etc. By taking Genzyme Corporation as a subsidiary, it added products like Cerezyme, Myozyme/Lumizyme & Fabrazyme to its portfolio. Sanofi has become a major player in the CHC sector. It acquired many companies i.e. Ablynx & Bioverativ, Synthorx & Principia & Translate Bio and Kadmon. It has collaborated with companies like Regeneron among others. It develops and markets Dupixent, Kevzara & Libtayo in collaboration with Regeneron. Sanofi, along with Regeneron, is now solely responsible for Praluent marketing in & outside the U.S. Sanofi operates through 3 Global Business Units: Specialty Care (neurology & immunology, rare diseases, rare blood disorders & oncology), Vaccines & General Medicines (diabetes, cardiovascular & established products) and Consumer Healthcare, a standalone unit.
Bluebird Bio (BLUE)Full Profile
bluebird bio, Inc. is a clinical-stage biotechnology that is focused on developing gene therapies for severe genetic diseases and cancer. The company has developed a deep pipeline using lentiviral-based gene therapies, T-cell immunotherapy expertise, and gene editing capabilities to treat severe genetic diseases as well as cancer.'The pipeline for severe genetic diseases includes betibeglogene autotemcel (beti-cel) for the treatment of transfusion-dependent '-thalassemia (TDT).
Abbvie Inc (ABBV)Full Profile
AbbVie has become one of the top-most pharma companies after it acquired Allergan. The deal has transformed AbbVie's portfolio by lowering its dependence on Humira, its flagship product. AbbVie has one of the most popular cancer drugs in its portfolio, Imbruvica and its newest immunology drugs Skyrizi and Rinvoq position it well for long-term growth. AbbVie came into existence after Abbott Laboratories divested its pharmaceutical division. AbbVie enjoys leadership positions in key therapeutic areas including immunology, hematologic oncology, neuroscience, aesthetics, eye care and womens' health. Humira is approved for several autoimmune diseases like rheumatoid arthritis, active psoriatic arthritis, active ankylosing spondylitis, Crohn's disease and others. Imbruvica became part of the company's portfolio following the Pharmacyclics acquisition. Other key drugs include Venclexta, Botox Cosmetic, Botox Therapeutics, Vraylar, Skyrizi and Rinvoq.
Abbott Laboratories (ABT)Full Profile
Abbott Labs discovers, develops, manufactures & sells a diversified line of health care products. Established Pharmaceuticals Division includes branded generics business in the emerging markets. Medical Devices unit includes the diabetes care, vision care & vascular businesses. Diagnostics unit manufactures and markets diagnostic systems and tests in four business lines core laboratory, molecular, point of care and rapid diagnostics. Nutrition unit includes a broad line of pediatric and adult nutritional products. Abbott acquired CFR Pharmaceuticals, Tendyne Holdings, Inc., St. Jude Medical and Alere Inc. On the other hand, Abbott sold its developed markets branded generics pharmaceuticals business to Mylan. Abbott retained the branded generics pharmaceuticals business in emerging markets. Abbott sold its animal health business to Zoetis Inc and divested its vision care business, Medical Optics, to Johnson & Johnson.
United Therapeutic (UTHR)Full Profile
United Therapeutics Corporation markets four medicines in the United States to treat pulmonary arterial hypertension (PAH): Remodulin, an injectable formulation of treprostinil, Orenitram, an oral version of treprostinil, Tyvaso, an inhaled version of treprostinil, and Adcirca (tadalafil; also sold by Eli Lilly as Cialis for erectile dysfunction) tablets.
Springworks Therapeutics Inc (SWTX)Full Profile
SpringWorks Therapeutics Inc. is a clinical-stage biopharmaceutical company. It is focused on developing life-changing medicines for patients with severe rare diseases and cancer. The company's product pipeline consists of Nirogacestat and Mirdametinib which are in clinical stage. SpringWorks Therapeutics Inc. is based in Stamford, United States.
Intercept Pharmaceuticals (ICPT)Full Profile
Intercept Pharmaceuticals, Inc. is a biopharmaceutical company focused on bringing novel therapeutics to treat chronic liver diseases to market. The company's focus is on treating orphan and more prevalent liver diseases with limited treatment options. The company received FDA approval for Ocaliva an farnesoid X receptor against in combination with ursodeoxycholic acid, for the treatment of primary biliary cholangitis in adults with an inadequate response to UDCA or as monotherapy in adults who are intolerant to UDCA. Ocaliva was approved in the EU for the same indication. The drug was approved in Canada. OCA is also being developed for additional indications like primary sclerosing cholangitis and non-alcoholic steatohepatitis. The FDA accepted its New Drug Application for OCA seeking accelerated approval for the treatment of fibrosis due to NASH. Intercept received a complete response letter from the FDA stating that its NDA for OCA for the treatment of liver fibrosis due to NASH could not be approved.