These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include:
Pipeline and key clinical candidates for these companies:
Rocket Pharmaceuticals is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The company's platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector-based gene therapy are for the treatment of Fanconi Anemia, a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I, a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency, a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket's first clinical program using adeno-associated virus-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.
Avidity Biosciences' proprietary AOCs - Antibody Oligonucleotide Conjugates - are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics, the company says. Avidity's advancing and expanding pipeline has three programs in clinical development. AOC 1001 is designed to treat people with myotonic dystrophy type 1 and is currently in Phase 1/2 development with the ongoing MARINA and MARINA-OLE trials. AOC 1020 is designed to treat people living with facioscapulohumeral muscular dystrophy and is currently in Phase 1/2 development with the FORTITUDE trial. AOC 1044 is designed for people with Duchenne muscular dystrophy mutations amenable to exon 44 skipping and is currently in Phase 1/2 development with the EXPLORE44 trial.
Hoth Therapeutics refers to itself as "a catalyst in early-stage pharmaceutical research and development, elevating drugs from the bench to pre-clinical and clinical testing." Hoth "collaborates and partners with a team of scientists, clinicians, and key opinion leaders to seek out and investigate therapeutics that hold immense potential to create breakthroughs and diversify treatment options," the company states.
Codexis is an enzyme engineering company leveraging its proprietary CodeEvolver technology platform to discover, develop and enhance novel, high-performance enzymes and other classes of proteins. "Codexis enzymes solve for real-world challenges associated with small molecule pharmaceuticals manufacturing, nucleic acid synthesis and genomic sequencing, and - as biotherapeutic candidates - they have the potential to treat challenging diseases," the company says.
Geron is a late-stage biopharmaceutical company pursuing therapies with the potential to extend and enrich the lives of patients living with hematologic malignancies. Its investigational first-in-class telomerase inhibitor, imetelstat, "harnesses Nobel Prize winning science in a treatment that may alter the underlying course of these diseases," the company says.
Neurocrine Biosciences describes itself as "a neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs, but few options." The company's portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, Parkinson's disease, endometriosis and uterine fibroids, as well as "a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas," the company states.
Axcella is "pioneering a new approach to treat complex diseases using compositions of endogenous metabolic modulators," or EMMs, the company states. The company's product candidates are comprised of EMMs and derivatives that are engineered in distinct combinations and ratios to reset multiple biological pathways, improve cellular energetics, and restore homeostasis. Axcella's pipeline includes lead therapeutic candidates in Phase 2 development for the treatment of Long COVID, NASH, and the reduction in risk of OHE recurrence.
Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA, or mRNA, to "an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio and integrated manufacturing facilities that allow for rapid clinical and commercial production at scale," the company states. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has "allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing." Most recently, Moderna's capabilities have come together to allow the authorized use and approval of vaccines against the COVID pandemic. The company is pursuing the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases.
Navidea Biopharmaceuticals is a biopharmaceutical company focused on the development of precision immunodiagnostic agents and immunotherapeutics. Navidea is developing multiple precision-targeted products based on its Manocept platform to enhance patient care by identifying the sites and pathways of disease and enable better diagnostic accuracy, clinical decision-making, and targeted treatment. The Manocept platform serves as the molecular backbone of Tc99m tilmanocept, the first product developed and commercialized by Navidea based on the platform.
Recent news on these stocks:
Neurocrine announced the FDA has accepted its New Drug Application - NDA - for Ingrezza oral granules, a new sprinkle formulation of Ingrezza capsules for oral administration. The agency set a Prescription Drug User Fee Act target action date of April 30, 2024. The Ingrezza oral granules capsules are intended to be opened for sprinkling on soft foods prior to administration. The NDA filing included chemistry, manufacturing, and controls information and data demonstrating the bioequivalence and tolerability of the Ingrezza oral granule sprinkle capsules compared to the currently approved Ingrezza capsules. Ingrezza is currently available as the only one-capsule, once-daily treatment option with no complex titration for adults with tardive dyskinesia and the treatment of chorea associated with Huntington's disease.
Neurocrine announced the FDA has accepted its New Drug Application, or NDA, for Ingrezza oral granules, a new sprinkle formulation of Ingrezza capsules for oral administration. The agency set a Prescription Drug User Fee Act target action date of April 30, 2024. The Ingrezza oral granules capsules are intended to be opened for sprinkling on soft foods prior to administration. The NDA filing included chemistry, manufacturing, and controls information and data demonstrating the bioequivalence and tolerability of the Ingrezza oral granule sprinkle capsules compared to the currently approved Ingrezza capsules. Ingrezza is currently available as the only one-capsule, once-daily treatment option with no complex titration for adults with tardive dyskinesia and the treatment of chorea associated with Huntington's disease.
Axcella Therapeutics announced that its board has approved a 1-for-25 reverse stock split of the company's common stock. The reverse stock split will become effective on September 18 after close of trading on The Nasdaq Global Market. The company's common stock is expected to commence trading on a split-adjusted basis when the markets open on September 19 under the existing trading symbol "AXLA."
Moderna announced that the European Medicines Agency's Committee for Medicinal Products for Human Use, or CHMP, has adopted a positive opinion recommending marketing authorization for Spikevax, its updated COVID-19 vaccine containing spike proteins for the XBB.1.5 sublineage of SARS-CoV-2 for active immunization to prevent COVID-19 caused by SARS-CoV-2 in individuals six months of age and older. Following the CHMP's positive opinion, the European Commission will make an authorization decision on the use of Moderna's updated COVID-19 vaccine for autumn/winter 2023, the company stated.
Rocket Pharmaceuticals' 7.81M share Spot Secondary priced at $16.00. The deal size was increased to $175M from $150M and priced at low end of its $16.00-$16.15 range. JPMorgan, Morgan Stanley, Leerink and TD Cowen are acting as joint book running managers for the offering.
Hoth Therapeutics announced that HT-KIT, a new molecular entity, for the treatment of advance systemic mastocytosis achieved positive preclinical results in a study sponsored by Hoth. HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frame shifting and already has Orphan Drug Designation from the FDA. The sponsored preclinical research was conducted at NC State University over the past two years. The aim of the initial studies was to assess how long HT-KIT was functional in cancerous and non-cancerous mast cells, and to determine whether the cells could develop resistance to HT-KIT. Key findings from initial studies in the mouse model show: HT-KIT effectively kills human mast cells that rely on signaling through the KIT receptor to survive; The effects of a single dose of HT-KIT lasted for about 2 weeks; HT-KIT efficacy at reducing KIT expression lasted for 7 days. Overall, these data provide strong support for potential applications of HT-KIT outside of mastocytosis and mast cell leukemia where KIT may be expressed and could play a role in disease aggression. Further in vivo and mechanistic studies are underway at NCSU in these diseases.
Goldman Sachs upgraded Geron to Buy from Neutral with an unchanged price target of $4. The analyst believes imetelstat is likely to be approved for the treatment of lower-risk myelodysplastic syndromes, where the firm estimates $1.5B in unadjusted peak sales. The recent approval of Reblozyl does not negate the blockbuster opportunity for imetelstat in the second-line setting, the analyst tells investors in a research note. Thus, Goldman sees the recent pullback in Geron shares as presenting a buying opportunity.
Codexis announced the company has executed an assignment and assumption of lease for its San Carlos, California location. As a result of consolidating operations to its headquarters facility in Redwood City, California, Codexis estimates it will realize cumulative cash savings of more than $30M through 2031. The execution of the agreement is consistent with the company's ongoing strategy to focus resources on programs with the strongest commercial opportunity and greatest probability of near-term success, including the company's enzyme-catalyzed oligonucleotide ECO Synthesis technology platform, designed to enable scalable production of RNA interference RNAi therapeutics. "The swift execution of this lease assignment demonstrates our ability to efficiently streamline the business and concentrate on the priorities we outlined in July as part of our enhanced strategic focus," said Kevin Norrett, MBA, COO of Codexis. "Having the entire company in one location will enable the close collaboration of our teams while also reducing overhead and positioning us to meet our projected cash runway to mid-2026."
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About "Biotech Alert"
The Fly will report on a selection of biotech stocks seeing a surge in interest from retail and financial professional investors, based on data from InvestingChannel.
This Fly exclusive recap reveals the biotech stocks that are seeing a spike in searches among the 20-plus million retail and financial professional investors through InvestingChannel's online financial news media ecosystem.
This increased attention from the investors may be in response to, or advance of, outsized moves for stocks in the biotech sector, which tend to be volatile and prone to sharp swings in share price around binary events such as clinical study results and FDA approvals.