Thank you for Subscribing to Life Science Review Weekly Brief
Governments, payers and consumers continue to place pressure on drug prices across the globe. In the U.S., pharmacy benefit managers (PBMs) and payers are negotiating more significant discounts, with declining net prices. Payers also are continuing to restrict coverage of therapies they deem too expensive; the Centers for Medicare & Medicaid Services’ (CMS) recent proposed coverage restriction of Aduhelm to clinical trial patients is just one example. Pricing pressure may also come from new competitors within the industry.
EQRx, the first of a potential wave of companies with disruptive “low-cost, me-too” strategies, intends to price its therapies 50-70 percent lower than competitors’ pricing. It has assembled a pipeline of at least five clinical assets and has partnerships or memoranda of understanding (MOUs) with CVS Health, Geisinger, Blue Shield of California, BlueCross BlueShield of North Carolina, Horizon and NHS England. The timing of its first filings remains to be seen, as their current pivotal trial data for two lead assets is from Chinese studies and U.S.- based studies are planned to begin this year. Additionally, the biosimilar pipeline is accelerating and may soon provide increased competition to numerous blockbuster franchises.
The Biden administration has set forth numerous proposed policies to control drug prices, such as Medicare drug price negotiation, penalties for price increases over inflation rates, earlier generic and biosimilar competition, and rebate transparency. Some of these provisions are included in the latest Build Back Better bill, though congressional gridlock makes it unclear whether and in what form they could be approved. Despite this, bipartisan pressure still exists for drug pricing reform, and smaller-scale changes in some form still could be imminent.
Combined, these trends are leading to fundamental shifts across the healthcare ecosystem. To win in this evolving landscape, industry leaders will have to monitor these trends and adapt their corporate strategies to make decisions regarding portfolio investments, new product launch strategies, capital formation and partnerships with industry participants.
We present to you, “Top 20 Clinical- Stage Biopharmaceutical Companies – 2022.”
AiViva has been developing novel, innovative therapies that can be delivered at the site of the disease to improve the effectiveness and decrease the side effects commonly seen for drugs taken orally. The company is dedicated to developing drug products with prolonged therapeutic effects—whose time of drug administration varies between three to six months. This reduces the treatment burden for both patients and physicians and improves the treatments’ benefit-risk profile.
AlaMab Therapeutics has emerged as a pioneer in developing novel, first-in-class antibody drugs. The company devotes a significant amount of time in educating the regulatory agency and their present and potential investors about their drug target. They aim to successfully launch this antibody and ensure that patients affordably have access to these drugs. The firm focuses on innovation and investing its resources more into the clinical and preclinical study of its current core product set and expanding indication portfolio.
Dr. Qingxi (Charles) Wang, ChairmanWe Aim to Successfully Launch These Antibodies and Ensure That Patients Have Access to These Drugs in an Affordable Way
Allakos is a clinical-stage company developing therapeutic antibodies for inhibitory receptors on the surface of immune effector cells involved in allergic, inflammatory, and proliferative diseases. Their lead drug candidate is lirentelimab (AK002), an investigational antibody that targets Siglec-8, an inhibitory receptor selectively found on mast cells and eosinophils. In clinical and preclinical studies, lirentelimab has been shown to broadly inhibit mast cells and rapidly deplete eosinophils.
Aprea Therapeutics, Inc. is a biopharmaceutical company headquartered in Boston, Massachusetts. Aprea is a Swedish biotech company focusing on the discovery and development of novel anticancer compounds reactivating the tumor suppressor protein p53. The Company's lead program, APR-246, is a first-in-class small molecule drug candidate in clinical development. The Company’s lead product candidate is APR-246 (eprenetapopt), a small molecule in clinical development for hematologic malignancies.
Biosion is a leading “In Global For Global” clinical stage biotechnology company developing innovative biologics to address resistant, relapsed and residual disease for patients worldwide. Established in 2017, Biosion has assembled a world-class team with extensive experience in global biologics development and built an oncology and auto-immune focused pipeline of innovative biologics through its internally-derived proprietary technologies, including the H3 hybridoma platform, SynAbTM synergistic antibody platform, SynTracerTM HT-endocytosis screening, and FlexibodyTM bispecific platforms.
Founded in late 2018 through a partnership with Pfizer and Bain Capital, Cerevel Therapeutics has a portfolio of experimental neuroscience therapies, which include three clinical-stage compounds and several pre-clinical compounds designed to target a broad range of CNS disorders, including Parkinson’s, Alzheimer’s, epilepsy, schizophrenia and addiction. The company has built a highly experienced team of senior leaders and neuroscience drug developers who combine a nimble, results-driven biotech mindset with the proven expertise of pharmaceutical company drug discovery and development.
Chinook Therapeutics's product candidates are being investigated in rare, severe chronic kidney disorders with opportunities for well-defined clinical pathways. It's lead program is atrasentan, a phase 3 endothelin receptor antagonist for the treatment of IgA nephropathy and other proteinuric glomerular diseases. Chinook is building its pipeline by leveraging insights in kidney single cell RNA sequencing, human-derived organoids and new translational models, to discover and develop therapeutics with differentiating mechanisms of action against key kidney disease pathways.
Citius Pharmaceuticals diversified pipeline of therapies targeting unmet medical needs includes three potential first-and-only prescription treatments in their indications, and a next-generation single-donor, allogeneic, clonal and scalable stem cell therapy program. Their team’s extensive experience in the pharmaceutical industry allows them to identify compelling opportunities and develop assets that have unique commercial advantages and differentiated upside potential. By deploying capital thoughtfully, they believe that their activities will provide patients and caregivers with improved treatment options and deliver long-term sustainable value to shareholders.
CohBar is a clinical stage biotechnology company. Mitochondria based therapeutics originate from the discovery by CohBar’s founders of a novel group of naturally occurring mitochondrial-derived peptides within the mitochondrial genome that regulate metabolism and cell death, and whose biological activity declines with age. To date, the company has discovered more than 100 mitochondrial derived peptides and generated over 1,000 analogs. CohBar’s efforts focus on the development of these peptides into therapeutics that offer the potential to address a broad range of diseases, including nonalcoholic steatohepatitis (NASH), obesity, fibrotic diseases, cancer, type 2 diabetes, and cardiovascular and neurodegenerative diseases.
Everest Medicines strives to be the partner of choice for innovative companies around the world with products that address critical unmet medical needs for patients in Greater China and have a high probability of market approval. Their team has deep expertise and extensive track record of high quality clinical development, regulatory affairs, CMC, business development and operations both in China and with leading global pharmaceutical companies. The company's Series A round was led by C-Bridge Capital, a top-tier China healthcare private equity firm, with US $800 million under management.
Eyenovia is a late-stage ophthalmic biopharmaceutical company developing breakthrough treatments for front and back-of-the-eye diseases, which are made possible by the company’s transformative, high precision ophthalmic delivery platform. Eyenovia’s pipeline is currently focused on the late-stage development of microdosed indications for presbyopia, myopia progression and mydriasis. The company believe that their innovation in ophthalmic delivery will change the way eyecare professionals and patients view eye medications. They strive to create drug therapies that are more effective, safer, and smarter than traditional eyedroppers
Fate Therapeutics has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Company’s immuno-oncology pipeline includes off-the-shelf, iPSC-derived natural killer (NK) cell and T-cell product candidates, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens using chimeric antigen receptors (CARs).
Hepion Pharmaceuticals's therapeutic approach targets fibrosis and hepatocellular carcinoma (HCC) associated with non-alcoholic steatohepatitis (NASH), viral hepatitis, and other liver diseases.Their cyclophilin inhibitor, CRV431, is being developed to offer benefits to address these multiple complex pathologies. The company's succes portfolio highligjhts their drug candidate which demonstrated good safety and tolerability in oral doses ranging from 75 mg to 525 mg. As expected, the pharmacokinetics of CRV431 showed increasing maximal blood concentrations as doses increased. Systemic blood exposures demonstrated good linearity up to 375 mg, which we anticipate will be more than sufficient for efficacy, based on our preclinical testing.
Karuna Therapeutics, through the cultivation of their deep expertise in neuroscience, and inventive thinking around drug discovery and development, they are advancing a pipeline of novel medicines with the goal of providing a meaningful difference in the lives of those affected by these conditions. The company sucess is defined by their passion to serve those affected by mental illness.
Prelude Therapeutics's lead product candidates are designed to be oral, potent, and selective inhibitors of PRMT5. Prelude’s first clinical candidate, PRT543, is in Phase 1 development for advanced solid tumors and select myeloid malignancies. Prelude is also advancing PRT811, a second PRMT5 inhibitor optimized for high brain exposure, in a Phase 1 clinical trial including glioblastoma multiforme. The Company’s pipeline also includes its third clinical candidate, PRT1419, an orally available MCL1 inhibitor in Phase 1 development for patients with relapsed/refractory hematologic malignancies, and its two most advanced preclinical candidates, PRT2527, a CDK9 inhibitor, and PRT-SCA2, a SMARCA2 protein degrader.
By leveraging our industry-leading core strength in CARD (Computer Accelerated Rational Discovery), Regor is committed to creating a highly efficient ecosystem for accelerated discovery of innovative therapeutic agents with differentiated therapeutic values for patients around the world. With a strong focus on excellence in pharmaceutical research and global competitiveness, they aspire to build a world-class innovation engine that promotes a winning culture of scientific excellence, strong teamwork driven by results and delivery with shared accountability and successes.
Relay Therapeutics is a clinical-stage precision medicines whose approach combines unprecedented computational power with leading-edge experimental approaches across the fields of structural biology, biophysics, chemistry and biology. By applying these insights, they utlizes a differentiated approach to drug protein targets based on their motion, enabling us to select and advance unique product candidates that can be developed into novel therapies for patients.
Spero Therapeutics is a multi-asset, clinical-stage biotech company focused on multidrug-resistant bacterial infections and rare disease. They are highly committed to advancing novel treatment approaches for bacterial infections with a world-class team of biotechnological and biopharmaceutical experts. The company has a pipeline of novel and highly differentiated antibacterial and rare disease product candidates focused on patients' unmet needs with multi-drug resistant (MDR) bacterial infections. They believe that their novel product candidates will have a meaningful impact on patient health and significant commercial applications for treating MDR infections in hospitals and community settings.
Verona Pharma is a clinical stage biopharmaceutical company currently focused on developing novel lead therapeutic candidate in the treatment of chronic obstructive pulmonary fibrosis (COPD) and cystic fibrosis (CF), and may expand our pipeline to help address the gaps in treatment of other respiratory diseases. The company has established relationships with leading research institutions and advocacy organizations in the respiratory space and have received awards in support of our continued work and innovation.
Zymeworks is a clinical-stage biopharmaceutical company dedicated to the development of next-generation multifunctional biotherapeutics. They operate with a mission to bring patients home to their loved ones, disease free. Zymeworks is advancing a deep preclinical pipeline in oncology (including immuno-oncology agents) and other therapeutic areas. In addition, its therapeutic platforms are being leveraged through strategic partnerships with nine biopharmaceutical companies.