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: 

• Palatin Technologies (PTN), 741% surge in interest
• Brainstorm Cell (BCLI), 42% surge in interest
• Cassava Sciences (SAVA), 37% surge in interest

Pipeline and key clinical candidates for these companies:

Palatin is a biopharmaceutical company developing medicines based on molecules that modulate the activity of the melanocortin receptor systems, with targeted, receptor-specific product candidates for the treatment of diseases with significant unmet medical need and commercial potential. “Palatin’s strategy is to develop products and then form marketing collaborations with industry leaders to maximize their commercial potential,” the company says.

BrainStorm Cell Therapeutics is a developer of autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the FDA and the EMA for the treatment of amyotrophic lateral sclerosis, ALS. BrainStorm has completed a Phase 3 pivotal trial in ALS; this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine.

Cassava Sciences is a clinical-stage biotechnology company that says its mission is to detect and treat neurodegenerative diseases, such as Alzheimer’s disease. “Its novel science is based on stabilizing-but not removing-a critical protein in the brain. The company’s product candidates have not been approved by any regulatory authority, and their safety, efficacy or other desirable attributes have not been established,” Cassava has stated.

Recent news on these stocks:

April 9

BrainStorm Cell Therapeutics received written agreement from the U.S. Food and Drug Administration, under a Special Protocol Assessment (SPA), on the design for a Phase 3b trial of NurOwn in amyotrophic lateral sclerosis. The SPA agreement with the FDA validates the clinical trial protocol and statistical analysis of the planned Phase 3b trial of NurOwn, demonstrating their adequacy for addressing objectives that support a future BLA in ALS. BrainStorm anticipates commencement of the Phase 3b study in 2024, after reviewing the protocol with investigators, securing study site Institutional Review Board approvals, and engaging with appropriate members of the ALS community. The Phase 3b trial will be a two-part, multicenter, study designed to assess the efficacy and safety of NurOwn in patients with ALS. The entry criteria will enroll participants earlier in the course of their disease, having the onset of ALS symptoms, including limb weakness, within the prior 24 months, all ALSFRS-R items 2 at Screening, and upright slow vital capacity greater than=65% of predicted for gender, height and age. Patients will also be allowed to receive concomitant treatment of an approved standard of care. Part-A is a double blind, placebo-controlled period of 24 weeks duration. Up to approximately 200 patients are planned to be enrolled and randomized 1:1 to NurOwn or placebo treatment groups. There will be a screening period of six to nine weeks, during which eligible participants will undergo a single bone marrow aspiration procedure to procure the mesenchymal stem cells that will be used to manufacture each participant’s NurOwn treatment for the duration of the trial. Patients will then be randomized 1:1 and treated with NurOwn or placebo via three repeated intrathecal injections, once every eight weeks. All eligible patients who complete Part-A will have the option of entering Part-B, open-label extension period of 24 weeks duration, where all participants will receive three repeated intrathecal injections of NurOwn, once every eight weeks. The primary efficacy endpoint is the change in the Revised Amyotrophic Lateral Sclerosis Functional Rating total score from baseline to Week 24. Primary inference from the trial will be based on a p-value from the combined assessment of function and survival to account for mortality observed in the trial. Cerebrospinal Fluid and blood samples will be collected for analysis of biomarkers of neuroinflammation, neurodegeneration, and neuroprotection. An independent Data Monitoring Committee will monitor the safety of the trial participants.

April 8

Palatin Technologies announced the presentation of topline results for its Phase 3 PL9643 MELODY-1 pivotal clinical trial evaluating the safety and efficacy of PL9643 versus vehicle in the treatment of dry eye disease at the American Society of Cataract and Refractive Surgery. The presentation by Dr. Eric Donnenfeld, entitled “Updates, What’s New in Treatment, What’s New and on the Threshold of FDA Approval” was presented on Friday, April 5th, at the ASCRS Conference in Boston, Massachusetts. Dr. Donnenfeld’s presentation included MELODY-1 Phase 3 clinical data results, which indicate that the Intent-to-Treat PL9643 treatment population demonstrated clinically meaningful and statistically significant results at the change from baseline to week 12 for the co-primary symptom endpoint of pain and multiple exploratory secondary symptom endpoints. The presentation also included an overview of the excellent and superior safety and tolerability profile of PL9643 compared to other approved treatments. “Analysis of our successful Phase 3 MELODY-1 clinical trial results, demonstrate that PL9643 has broad, robust and rapid efficacy for multiple symptom endpoints. The efficacy results were statistically significant for the co-primary symptom endpoint of pain and 7 of 11 exploratory secondary endpoints, including eye dryness, as early as two weeks (the 1st time point measured). The effect improved and maintained statistical significance over the 12-week treatment period,” said Carl Spana, Ph.D., President and Chief Executive Officer of Palatin. “Additionally, we have identified a substantial patient population with statistically significant efficacy results after two weeks of treatment with PL9643 for multiple sign endpoints, including all four fluorescein staining endpoints, which improves ocular surface disorders and facilitates the identification and treatment of epithelial damage and corneal injuries.” “The early onset of efficacy for multiple symptoms and signs of dry eye disease and the excellent ocular safety and tolerability profile positions PL9643 as a highly differentiated product. We are advancing discussions with potential collaboration partners and actively preparing for a meeting with the FDA later this quarter to discuss the remaining studies for the PL9643 program required to support an NDA submission,” Dr. Spana continued. “Dr. Donnenfeld’s presentation is important, not only because it illustrates PL9643 MELODY-1 positive results in the context of other developmental programs for DED but identifies a potential key differentiating treatment option for dry eye disease in terms of its safety and tolerability profile,” commented Michael Raizman, M.D., Chief Medical Officer at Palatin. Safety analysis from the Phase 3 MELODY-1 trial indicated PL9643 was well-tolerated. There were fewer ocular treatment related adverse events in the PL9643 arm compared to vehicle, and fewer study discontinuations in the PL9643 arm compared to vehicle. A higher proportion of the vehicle-treated patients dropped out of the study prior to week 12 compared to the PL9643-treated patients.

Hear more from InvestingChannel by signing up for The Spill.

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.