Biotech Alert: Searches spiking for these stocks today

These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include: 

• Avidity Biosciences (RNA), 4,591% surge in interest
• Argenx (ARGX), 933% surge in interest
• CohBar (CWBR), 864% surge in interest
• iBio (IBIO), 684% surge in interest
• RedHill Biopharma (RDHL), 551% surge in interest
• Springworks Therapeutics (SWTX), 547% surge in interest
• Cabaletta Bio (CABA), 546% surge in interest
• Seelos Therapeutics (SEEL), 252% surge in interest
• Arrowhead Pharma (ARWR), 200% surge in interest
• Journey Medical (DERM), 193% surge in interest

Pipeline and key clinical candidates for these companies:

Avidity Bioscience’s 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.

Argenx is an immunology company that says it is “committed to improving the lives of people suffering from severe autoimmune diseases” and “aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines.” Argenx developed and is commercializing what it calls “the first-and-only approved neonatal Fc receptor blocker in the U.S., the EU and UK, and Japan.” The company is evaluating efgartigimod in multiple autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises.

CohBar is a clinical-stage biotechnology company leveraging the power of the mitochondria and the peptides encoded in its genome to develop potential breakthrough therapeutics targeting chronic and age-related diseases with limited to no treatment options.

iBio develops next-generation biopharmaceuticals using computational biology and 3D-modeling of subdominant and conformational epitopes, prospectively enabling the discovery of new antibody treatments for hard-to-target cancers and other diseases. iBio’s mission is to “decrease drug failures, shorten drug development timelines, and open up new frontiers against the most promising targets,” the company stated.

RedHill Biopharma is primarily focused on gastrointestinal and infectious diseases. RedHill promotes the gastrointestinal drugs, Movantik for opioid-induced constipation in adults, Talicia for the treatment of Helicobacter pylori infection in adults, and Aemcolo for the treatment of travelers’ diarrhea in adults.

SpringWorks is a commercial-stage biopharmaceutical company applying a precision medicine approach to developing and delivering life-changing medicines for people with severe rare diseases and cancer. Ogsiveo, approved in the United States for the treatment of adult patients with progressing desmoid tumors who require systemic treatment, is SpringWorks’ first FDA-approved therapy.

Caballeta Bio is focused on the discovery and development of engineered T cell therapies that have the potential to provide a deep and durable, perhaps curative, treatment for patients with autoimmune diseases. The CABA platform encompasses chimeric antigen receptor T cells for autoimmunity and Cabaletta Bio’s proprietary chimeric autoantibody receptor T cells.

Seelos Therapeutics is focused on the development and advancement of novel therapeutics to address unmet medical needs for the benefit of patients with central nervous system, or CNS, disorders and other rare diseases. The company’s portfolio includes several late-stage clinical assets targeting indications including Acute Suicidal Ideation and Behavior in Major Depressive Disorder, amyotrophic lateral sclerosis, or ALS, and spinocerebellar ataxia, or SCA, as well as early-stage programs in Huntington’s disease, Alzheimer’s disease, and Parkinson’s disease.

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

Journey Medical says it is focused on identifying, acquiring, developing and strategically commercializing “innovative, differentiated dermatology products through its efficient sales and marketing model.” The company currently markets eight branded and three generic products that help treat and heal common skin conditions.

Recent news on these stocks:

November 28

Avidity Biosciences announced a global licensing and research collaboration with Bristol Myers Squibb (BMY) focused on the discovery, development and commercialization of multiple cardiovascular targets with potential cumulative payments of up to $2.3B. Antibody Oligonucleotide Conjugates, or AOCs, 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. This strategic collaboration broadens the reach of AOCs through the expansion of the existing relationship with Bristol Myers Squibb. Avidity continues to advance its internal research and development programs in rare cardiac indications. Under the terms of the agreement, Avidity will receive $100M upfront, which includes a $60M cash payment as well as the purchase of approximately $40M of Avidity common stock at a purchase price of $7.88 per share. Avidity is also eligible to receive up to approximately $1.35B in research and development milestone payments, up to approximately $825M in commercial milestone payments, and tiered royalties up to low double-digits on net sales. Bristol Myers Squibb will fund all future clinical development, regulatory and commercialization activities coming from this collaboration.

Argenx announced topline results from the ADVANCE-SC study evaluating Vyvgart Hytrulo in adults with primary immune thrombocytopenia, or ITP. The study did not meet the primary endpoint of a sustained platelet count response in chronic ITP patients. Additional analyses of the dataset are ongoing and the full results will be presented at an upcoming medical meeting and in a peer-reviewed publication. ADVANCE-SC is the second of two registrational trials conducted as part of the ongoing ITP development program for Vyvgart and enrolled 207 adult patients with chronic and persistent ITP. Patients were heavily pre-treated and 75% of patients had received three or more prior ITP therapies. Primary endpoint was not met; 13.7% of treated patients demonstrated a sustained platelet count response compared to 16.2% of placebo patients. Secondary endpoints were not met, including additional endpoints on International Working Group, or IWG, responder status and mean platelet count change from baseline. Vyvgart Hytrulo was well-tolerated in ADVANCE-SC; the observed safety and tolerability profile was consistent with ADVANCE-IV and the confirmed safety profile of Vyvgart and Vyvgart Hytrulo. Results from the first study in the ITP registrational program, ADVANCE-IV, were reported in May 2022. The study met its primary and key platelet-derived secondary endpoints. ADVANCE-IV formed the basis of the regulatory submission for approval of Vyvgart IV for ITP in Japan, where a decision is expected in the first quarter of 2024. Vyvgart is currently being evaluated in 13 severe autoimmune diseases, including the registrational ADDRESS study for pemphigus from which topline results are expected around year-end 2023.

Cantor Fitzgerald wrote in a research note that Cabaletta Bio and other stocks were down following news that the FDA is investigating the link between CAR-T and T-cell malignancies with approved products in oncology. While the report might slow down progress at some centers, this is “likely a storm in a tea cup,” as INDs are still being approved, trials are still proceeding, unmet need remains high, the analyst tells investors in a research note. The firm, which made no change to its Overweight rating or $50 price target on Cabaletta Bio, is not going to be spooked by headlines that say the “FDA is looking into this,” as it has already accepted many development risks to its thinking.

Arrowhead Pharmaceuticals announced that it has filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-DM1, the company’s investigational RNA interference RNAi therapeutic being developed as a potential treatment for type 1 myotonic dystrophy DM1 the most common adult-onset muscular dystrophy. ARO-DM1 is designed to reduce expression of the dystrophia myotonica protein kinase DMPK gene. There is currently no approved disease-modifying therapy for DM1. Treatments have focused on symptomatic management, including physical therapy, exercise, ankle-foot orthoses, wheelchairs, and other assistive devices. James Hamilton, M.D., MBA, Chief of Discovery and Translational Medicine at Arrowhead, said: “ARO-DM1 is Arrowhead’s second clinical candidate utilizing our TRiM platform to deliver RNAi therapeutics to skeletal muscle. Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities and may become physically disabled and have a shortened life expectancy. ARO-DM1 represents a novel approach to treat DM1 by silencing aberrantly transcribed DMPK mRNA, which could lead to improvements in multiple symptoms, including muscle strength and function.” An application for approval of the clinical trial was submitted to a local Ethics Committee and to the New Zealand Medicines and Medical Devices Safety Authority for review by the Standing Committee on Therapeutic Trials. Pending clearance, Arrowhead intends to proceed with ARODM1-1001, a Phase 1/2a dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-DM1 in up to 48 subjects with DM1.

November 27

iBio announced that its board has approved a 20-to-1 reverse split of the company’s common stock, effective at 12:01 a.m. ET on November 29. The common stock is expected to begin trading on a split-adjusted basis when the market opens on November 29.

RedHill Biopharma announced that the FDA has granted five years’ market exclusivity for Talicia under the Generating Antibiotic Incentives Now Act Qualified Infectious Disease Product designation, as recommended by the FDA Exclusivity Board. This grant is on top of three years’ exclusivity granted for the approval of Talicia under section 505(b)(2). Talicia is protected by its broad intellectual property suite to 2034. “Talicia is the only FDA-approved rifabutin-based therapy for the eradication of H. pylori. Its components and formulation are optimized to provide patients with the necessary medications for successful H. pylori eradication, an innovation rewarded by this market exclusivity and other IP, providing protection to 2034,” said Patricia Anderson, RedHill’s senior VP of Regulatory Affairs. “With significant concerns around maintaining effective therapeutic options for infectious diseases in the face of growing microbial resistance, it is vital that the most effective options are utilized first line. Talicia’s favorable efficacy, tolerability, and resistance profile, in the only all-in-one formulation available, provides potential advantages over clarithromycin-based regimens for most patients.” The FDA recently approved a supplemental new drug application for Talicia, allowing a change to a more flexible three times daily, taken at least 4 hours apart with food, dosing regimen for H. pylori eradication enabling patients to follow a convenient “breakfast, lunch and dinner” dosing routine, which may support increased patient adherence and optimize the potential for successful H. pylori eradication.

SpringWorks Therapeutics announced that the FDA has approved OGSIVEO, an oral gamma secretase inhibitor, for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. The FDA previously granted breakthrough therapy, fast track and orphan drug designations to nirogacestat for the treatment of desmoid tumors.

Seelos Therapeutics announced that SLS-002 has been selected for inclusion in an adaptive platform trial to evaluate treatments for post-traumatic stress disorder in active-duty service members and veterans. The trial is funded by the U.S. Department of Defense’s Defense Health Agency and led by the Warfighter Readiness, Performance, and Brain Health Project Management Office, part of the U.S. Army Medical Materiel Development Activity. “Our inclusion in this landmark adaptive platform study, funded by the U.S. Department of Defense, allows us to expedite pursuing indications beyond our current work in acute suicidal ideation and behavior in major depressive disorder,” said Raj Mehra, Ph.D., Chairman and CEO of Seelos. The Department of Defense PTSD Adaptive Platform Trial is a Phase II randomized, double-blinded, placebo-controlled study that will evaluate the safety, tolerability, and efficacy of multiple pharmacotherapeutic interventions in active-duty service members and veterans with PTSD. The trial will utilize an adaptive platform trial design randomizing participants among the multiple treatment cohorts selected for inclusion in the study and enable sharing of control participants to increase study efficiency. The design of the trial entails a 30-day screening period, a 12-week treatment period and a 4-week safety follow-up and will collect data to measure changes in PTSD symptom severity, as measured by the Clinician-Administered PTSD Scale-5-Revised and other clinically relevant endpoints, including the incidence of new or worsening suicidal thoughts or behaviors, as measured by change in the Columbia Suicide Severity Rating Scale score. In addition, the trial will evaluate several biomarkers associated with PTSD and assessments of treatment safety and tolerability.

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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.