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), 1,002% surge in interest
• Leap Therapeutics (LPTX), 226% surge in interest
• Brainstorm Cell (BCLI), 215% surge in interest
• Cassava Sciences (SAVA), 212% surge in interest
• Eyenovia (EYEN), 211% surge in interest
• Cue Biopharma (CUE), 141% surge in interest
• Molecular Templates (MTEM), 120% surge in interest
• VBI Vaccines (VBIV), 114% surge in interest
• Syros Pharmaceuticals (SYRS), 109% surge in interest
• Alkermes (ALKS), 106% 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.

Leap Therapeutics is focused on developing targeted and immuno-oncology therapeutics. Leap’s most advanced clinical candidate, DKN-01, is a humanized monoclonal antibody targeting the Dickkopf-1 protein. DKN-01 is being developed in patients with esophagogastric, gynecologic, and colorectal cancers. Leap has entered into a strategic collaboration with BeiGene (BGNE) for the rights to develop DKN-01 in Asia excluding Japan, Australia, and New Zealand.

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.

Eyenovia is an ophthalmic pharmaceutical technology company developing a pipeline of microdose array print therapeutics. Eyenovia is currently focused on the late-stage development of microdosed medications for mydriasis, presbyopia, and myopia progression.

Cue Biopharma, a clinical-stage biopharmaceutical company, is developing a novel class of injectable biologics to selectively engage and modulate disease-specific T cells directly within the patient’s body. The company’s proprietary platform, Immuno-STAT, and biologics are “designed to harness the body’s intrinsic immune system as T cell engagers without the need for ex vivo manipulation or broad systemic modulation,” Cue says.

Molecular Templates is a clinical-stage biopharmaceutical company focused on the discovery and development of targeted biologic therapeutics. The company’s proprietary drug platform technology, known as engineered toxin bodies, or ETBs, leverages the resident biology of a genetically engineered form of Shiga-like Toxin A subunit to create novel therapies with potent and differentiated mechanisms of action for cancer.

VBI Vaccines is a biopharmaceutical company focused on virus-like particles, or VLPs, including a proprietary enveloped VLP, eVLP, platform technology. VBI develops vaccine candidates that mimic the natural presentation of viruses, designed to elicit the innate power of the human immune system. “VBI is committed to targeting and overcoming significant infectious diseases, including hepatitis B, coronaviruses, and cytomegalovirus, as well as aggressive cancers including glioblastoma,” the company says.

Syros is committed to developing new standards of care for the frontline treatment of patients with hematologic malignancies. Driven by the motivation to help patients with blood disorders that have largely eluded other targeted approaches, Syros is developing tamibarotene, an oral selective RARalpha agonist in frontline patients with higher-risk myelodysplastic syndrome and acute myeloid leukemia with RARA gene overexpression.

Alkermes is a fully-integrated, global biopharmaceutical company developing innovative medicines in the fields of neuroscience and oncology. The company has a portfolio of proprietary commercial products focused on alcohol dependence, opioid dependence, schizophrenia and bipolar I disorder, and a pipeline of product candidates in development for neurological disorders and cancer.

Recent news on these stocks:

April 11

Leap Therapeutics announced it has entered into a securities purchase agreement with a select group of institutional investors to issue and sell an aggregate of 12.66M shares of its common stock at a price of $2.82 per share and pre-funded warrants to purchase 1.52M shares of common stock at a price of $2.819 per share of common stock issuable upon exercise of the pre-funded warrants, in a private placement. Leap anticipates the gross proceeds from the private placement will be approximately $40M, before deducting any offering-related expenses. The financing is expected to close on April 15, subject to satisfaction of customary closing conditions. The private placement investors included new and existing investors, including Gilead Sciences, Samsara BioCapital, 683 Capital Partners, LP, Laurion Capital Management, and Rock Springs Capital. J.P. Morgan acted as the exclusive placement agent for the transaction.

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.

Molecular Templates announced that will present a poster, “First-in-human, dose escalation and expansion study of MT-6402, a novel engineered toxin body ETB targeting PD-L1, in patients with PD-L1 expressing relapsed/refractory advanced solid tumors: Interim Data,” at the 2024 American Association for Cancer Research Annual Meeting taking place in San Diego, CA, and also announced monotherapy response activity in Head and Neck Cancer…”We are excited to see objective responses in heavily pre-treated, checkpoint-experienced, head and neck cancer patients, a setting with high unmet medical need,” said CEO Eric Poma. “The long-lasting partial responses were in patients with low PD-L1 expression and showed concomitant increases in cytokines associated with T-cell activation and tumor microenvironment remodeling not seen with other checkpoint therapies. We believe the novel mechanism of action of MT-6402 may allow for durable, T-cell-mediated, monotherapy activity in patients who have progressed on checkpoint therapy. Based on the preliminary signals of single agent activity with MT-6402, an expansion study in low PD-L1 head and neck cancer patients to further evaluate MT-6402 has been initiated.”

VBI Vaccines announced that it has entered into definitive agreements for the sale and issuance of 2.27M common shares or pre-funded warrants in lieu thereof of VBI at an offering price of 88c per common share or per pre-funded warrant in lieu thereof in a registered direct offering priced at-the-market under the Nasdaq rules. In a concurrent private placement, VBI will issue unregistered warrants to purchase up to 2.27M common shares. The warrants have an exercise price of 76c per share, will be exercisable on the date of issuance, and will expire five years following the date of issuance. The closing of the offering is expected to occur on or about April 11, subject to the satisfaction of customary closing conditions. H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering. The gross proceeds to VBI from this offering are expected to be approximately $2M, which VBI intends to use for working capital and general corporate purposes.

Syros Pharmaceuticals announced that the FDA has granted Fast Track Designation, FTD, to tamibarotene in combination with azacitidine and venetoclax for the treatment of newly diagnosed acute myeloid leukemia with RARA overexpression as detected by an FDA approved test in adults who are over age 75 years or who have comorbidities that preclude the use of intensive induction chemotherapy. “We are pleased to receive Fast Track designation for tamibarotene for the treatment of AML. This designation reflects the tremendous need for a safe and effective therapy, which can improve the clinical outcomes and prognosis among people diagnosed with AML, many of whom cannot tolerate intensive treatment,” said David Roth, M.D., Chief Medical Officer of Syros Pharmaceuticals. “We are particularly encouraged to secure Fast Track designation following initial randomized data from a prespecified interim analysis of our ongoing SELECT-AML-1 clinical trial, in which treatment with our RARalpha agonist, tamibarotene, in combination with venetoclax and azacitidine resulted in a 100% CR/CRi rate compared with a 70% CR/CRi rate for the comparator of venetoclax and azacitidine. Additionally, tamibarotene in combination with venetoclax and azacitidine demonstrated no added toxicity relative to venetoclax and azacitidine alone. We look forward to sharing additional data from SELECT-AML-1 later this year, and to potentially accelerate the delivery of tamibarotene as a new frontline option for the approximately 30% of AML patients who are positive for RARA overexpression.”

Alkermes announced positive topline results from the narcolepsy type 2 – NT2 – and idiopathic hypersomnia – IH – cohorts of a phase 1b, proof-of-concept study evaluating ALKS 2680, the company’s novel, investigational, oral orexin 2 receptor agonist in development as a once-daily treatment for narcolepsy. ALKS 2680 data demonstrated clinically meaningful and statistically significant improvements from baseline in mean sleep latency on the Maintenance of Wakefulness Test vs. placebo at all doses tested. ALKS 2680 was generally well tolerated in both patient populations at all doses tested. The company plans to initiate a phase 2 study in patients with NT2 in the second half of 2024. Alkermes expects to submit results from this phase 1b, proof-of-concept study to a peer-reviewed journal for publication and to present additional ALKS 2680 study results at upcoming scientific meetings.

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.

Eyenovia announced its plans for accelerating development of its potential multi-billion dollar product for pediatric progressive myopia, MicroPine, and initiated a process to explore strategic alternatives to maximize shareholder value. As part of this process, the Company plans to consider a wide range of options, including potential sale of assets of the Company, a sale of the Company, a merger, or other strategic action. In conjunction with the strategic process and focus on its late stage MicroPine asset, Eyenovia reiterated its immediate commercialization opportunities and corporate savings intended to reduce operating expenses while continuing to support meaningful value generation from the company’s two FDA-approved products. …”In the meantime, we will be evaluating strategic opportunities for MicroPine with organizations that have existing capabilities and infrastructure necessary to fully capitalize on this large opportunity. Additionally, we continue to make significant progress advancing our commercialization strategy, with Mydcombi and Avenova currently being sold through our targeted sales force, and clobetasol to be commercially launched as soon as this summer.”…”However, we believe this progress, and the broader potential of MicroPine, which will be back in our hands in a matter of weeks, has been underrecognized and underappreciated by the financial markets. As a result, while we remain intently focused on laying a foundation for accelerating sales growth in 2025, we are in parallel reducing costs in areas that do not impact our ability to deliver on our core strategy and exploring a possible transaction or transactions that could generate capital or otherwise enhance value for our shareholders, who remain very supportive. I look forward to evaluating a comprehensive variety of alternatives with my fellow Board members in the coming weeks.” …Eyenovia is in the process of engaging with investment banks to assist with the evaluation. There can be no assurance that this process will result in the company entering or completing any transaction. Eyenovia does not intend to make any further disclosures regarding the strategic review process unless and until a specific course of action is approved.

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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.