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: 

• Aldeyra (ALDX), 1,642% surge in interest
• ESSA Pharma (EPIX), 1,550% surge in interest
• Mereo Biopharma (MREO), 1,162% surge in interest
• Prothena (PRTA), 1,092% surge in interest
• ContraFect (CFRX), 424% surge in interest
• Omeros Corp (OMER), 397% surge in interest
• Eloxx Pharmaceuticals (ELOX), 231% surge in interest
• Bellerophon (BLPH), 172% surge in interest
• Applied Therapeutics (APLT), 167% surge in interest
• BioNTech (BNTX), 152% surge in interest

Pipeline and key clinical candidates for these companies:

Aldeyra is a clinical-stage biotechnology company whose pre-commercial product candidates are reproxalap, a potential treatment for dry eye disease and allergic conjunctivitis, and ADX-2191, a potential treatment for primary vitreoretinal lymphoma, proliferative vitreoretinopathy, and other rare sight-threatening retinal diseases. In addition, Aldeyra is developing other product candidates, including ADX-629 and chemically related molecules, for the potential treatment of systemic and retinal immune-mediated diseases.

ESSA is focused on developing novel and proprietary therapies for the treatment of patients with prostate cancer. ESSA is developing the first generation of ANITen bAsed Chimera degraders targeting the AR NTD. In preclinical models, the orally bioavailable ANITAC degraders can eliminate forms of AR protein found in castration-resistant prostate cancer that can potentially drive disease progression including LBD mutants and LBD truncated splice variants.

Mereo BioPharma is a biopharmaceutical company focused on the development of innovative therapeutics for rare diseases. The company has two rare disease product candidates, setrusumab for the treatment of Osteogenesis Imperfecta (OI) and alvelestat for the treatment of severe alpha-1-antitrypsin deficiency-associated lung disease (AATD-LD) and Bronchiolitis Obliterans Syndrome (BOS).

Prothena Corporation is a late-stage clinical biotechnology company with expertise in protein dysregulation and a pipeline of investigational therapeutics with the potential to change the course of devastating neurodegenerative and rare peripheral amyloid diseases. Fueled by its deep scientific expertise built over decades of research, Prothena is advancing a pipeline of therapeutic candidates for a number of indications and novel targets for which its ability to integrate scientific insights around neurological dysfunction and the biology of misfolded proteins can be leveraged. Prothena’s pipeline includes both wholly-owned and partnered programs being developed for the potential treatment of diseases including AL amyloidosis, ATTR amyloidosis, Alzheimer’s disease, Parkinson’s disease and a number of other neurodegenerative diseases.

ContraFect is a biotechnology company focused on the discovery and development of DLAs, including lysins and amurin peptides, as new medical modalities for the treatment of life-threatening, antibiotic-resistant infections. The company has completed a Phase 2 clinical trial for the treatment of Staph aureus bacteremia, including endocarditis, with its lead lysin candidate, exebacase, which is the first lysin to enter clinical studies in the U.S. Exebacase was granted Breakthrough Therapy designation by the FDA for the treatment of MRSA bloodstream infections, including right-sided endocarditis, when used in addition to SOC anti-staphylococcal antibiotics.

Omeros is committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market and orphan indications targeting immunologic disorders, including complement-mediated diseases, cancers, and addictive and compulsive disorders. Omeros’ lead MASP-2 inhibitor narsoplimab targets the lectin pathway of complement and is the subject of a biologics license application pending before FDA for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy. Narsoplimab is also in multiple late-stage clinical development programs focused on other complement-mediated disorders, including IgA nephropathy, COVID-19, and atypical hemolytic uremic syndrome.

Eloxx Pharmaceuticals’ lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. It is in clinical development, focusing on cystic fibrosis. The FDA has granted Fast Track designation for ELX-02 for the treatment of CF patients with nonsense mutations. In addition, ELX-02 has also been granted Orphan Drug Designation for the treatment of CF patients with nonsense mutations by the FDA and orphan medicinal product designation by the European Commission.

Bellerophon Therapeutics is focused on developing therapies that address significant unmet medical needs in the treatment of cardiopulmonary diseases. The company is currently developing multiple product candidates under its INOpulse program, a proprietary pulsatile nitric oxide delivery system.

Applied Therapeutics says it is “developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need.” The company’s lead drug candidate, AT-007, is a novel central nervous system penetrant Aldose Reductase Inhibitor for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG. The company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart.

BioNTech is “pioneering novel therapies for cancer and other serious diseases.” BioNTech and its collaborators are developing multiple mRNA vaccine candidates for a range of infectious diseases alongside its oncology pipeline. BioNTech has established a broad set of relationships with multiple global pharmaceutical collaborators, including Genmab (GMAB), Sanofi (SNY), Genentech, a member of the Roche Group (RHHBY), Regeneron (REGN), Genevant, Fosun Pharma, and Pfizer (PFE).

Recent news on these stocks:

October 16

In a regulatory filing, the company stated: “On October 16, 2023, Aldeyra Therapeutics announced that it received minutes from a late-cycle review meeting with the U.S. Food and Drug Administration relating to the new drug application for reproxalap for the treatment of the signs and symptoms of dry eye disease. The Minutes identified substantive review issues in connection with the NDA for reproxalap. The FDA stated that “(i)t does not appear that you have data to support the clinical relevance of the ocular signs to support your dry eye indication.” In subsequent communications between Aldeyra and the FDA, Aldeyra has submitted responses to the FDA that Aldeyra believes to be sufficient to mitigate the identified issues, but the FDA has not directly opined on the sufficiency of the information submitted, has no legal obligation to review the information submitted by Aldeyra, and has indicated that Aldeyra needs to conduct an additional clinical trial to satisfy efficacy requirements. As such, based on the time remaining in the NDA review cycle, the FDA may not be in the position to approve the NDA for reproxalap on or about the Prescription Drug User Fee Act target action date of November 23, 2023 or afterwards, and it may issue a Complete Response Letter and require that Aldeyra conduct additional clinical trials and submit the results of those clinical trials before the application will be reconsidered.”

Prothena is preparing for a potential sale ahead of key Alzheimer’s data expected in the coming months, Michelle Davis, Dinesh Nair and Manuel Baigorri of Bloomberg report, citing people with knowledge of the matter. The company has been speaking to advisers as it gets ready to explore strategic options that may include a sale or partnership, sources told Bloomberg. The people added that Prothena would likely attract interest from large drugmakers. Deliberations are at an early stage, and Prothena could opt to remain independent for longer, the sources added.

ContraFect announces that the U.S. Food and Drug Administration, FDA, has notified the company that it has completed the safety review of its Investigational New Drug, IND, application for CF-370 for the treatment of hospital-acquired bacterial pneumonia, HABP, and ventilator-associated bacterial pneumonia, VABP, and concluded that the company may proceed with its Phase 1 clinical study. This milestone is significant for ContraFect and historic in the field of non-traditional antibacterial therapies, as CF-370 will be the first engineered lysin therapeutic targeting Gram-negative pathogens to enter a human clinical trial. CF-370 has demonstrated potent in vitro activity against Pseudomonas aeruginosa, Acinetobacter baumannii, Klebsiella pneumoniae, Escherichia coli and Enterobacter cloacae and across numerous animal models of pneumonia, including efficacy against multi-drug resistant strains. ContraFect is a global innovator in the discovery and development of DLAs, leading the field with an intellectual property portfolio which includes thirty U.S. patents, one hundred and fifty-four issued foreign patents and two hundred and forty-six pending U.S. and international patent applications. The company’s discovery platform and functional expression library are designed to enable the construction and identification of novel therapeutic proteins that can penetrate the outer membrane of Gram-negative bacteria, resulting in lysis and prokaryotic cell death. CF-370 represents the first product candidate to be developed using the company’s proprietary technologies.

Omeros provided an update regarding the interim analysis outcome in ARTEMIS-IGAN, the company’s Phase 3 trial evaluating narsoplimab for the treatment of immunoglobulin A, or IgA nephropathy. The primary endpoint is reduction in proteinuria assessed by 24-hour urine protein excretion at 36 weeks compared to placebo in the intent-to-treat population of 180 IgA nephropathy patients with high baseline proteinuria. Topline results show that narsoplimab did not achieve statistically significant improvement over placebo. The UPE reduction in the placebo group was markedly greater than that reported in trials of other agents in IgA nephropathy. Based on the absence of statistical significance and as previously agreed with FDA, Omeros will not submit an application for approval of narsoplimab in this indication and will discontinue the ARTEMIS-IGAN clinical trial.

October 15

Ultragenyx Pharmaceutical (RARE) and Mereo BioPharma announced interim data from the Phase 2 portion of the Phase 2/3 Orbit study demonstrating that treatment with setrusumab significantly reduced incidence of fractures in patients with OI with at least 6 months of follow-up and continues to demonstrate ongoing and meaningful improvements in lumbar spine bone mineral density. The data were presented in a late-breaker presentation at the American Society for Bone and Mineral Research 2023 Annual Meeting. As of the cut-off date and following at least 6 months of treatment with setrusumab, the annualized fracture rate across all 24 patients in the Phase 2 portion of the study was reduced by 67%. In the 2 years prior to treatment with setrusumab all patients experienced at least 1 fracture. The median annualized fracture rate of 0.72 in the 2 years prior to treatment was reduced to 0.00 during the mean treatment duration period of 9 months. Following initiation of treatment with setrusumab, 20 patients experienced no radiographic-confirmed fractures, and 4 patients experienced 7 radiographic-confirmed fractures in 5 separate events. These fractures exclude fractures of the fingers, toes, skull, and face consistent with the Phase 3 study design. The reduction in annualized fracture rates was associated with a clinically meaningful increase in BMD. At the 6-month timepoint, treatment with setrusumab resulted in a mean increase in lumbar spine BMD from baseline of 13% at 20 mg/kg and 16% at 40 mg/kg, which represents the same substantial mean improvement in Z-score of +0.85 for both dose groups at 6 months compared to a combined mean baseline Z-score of -1.68. The small apparent difference in BMD change from baseline is likely related to differences in patients assigned to the two treated groups. There was no statistically significant difference in BMD percent change or Z-score change from baseline between the 20 and 40 mg/kg dosing cohorts. As of the data cut-off, there were no treatment-related serious adverse events observed in the study. Reported adverse events were generally consistent with those observed in the ASTEROID study with infusion-related events and headache determined to be the most common adverse events related to the study drug. There have been no reported hypersensitivity reactions related to setrusumab. There were no notable safety-related differences observed between dosing groups or age groups.

October 12

Theodore Wang notified the board of Bellerophon Therapeutics of his resignation as a member of the company’s board, effective immediately. The resignation of Wang from the board was due to a disagreement as to whether the company’s board should approve the plan of liquidation and dissolution of the company.

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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.