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: 

• Cellectis (CLLS), 2,180% surge in interest
• Leap Therapeutics (LPTX), 883% surge in interest
• Redhill Biopharma (RDHL), 567% surge in interest
• PDS Biotechnology (PDSB), 505% surge in interest
• Syndax Pharma (SNDX), 105% surge in interest
• Axsome Therapeutics (AXSM), 102% surge in interest
• Cellectar Biosciences (CLRB), 100% surge in interest
• Novavax (NVAX), 75% surge in interest
• Intellia Therapeutics (NTLA), 62% surge in interest
• CRISPR Therapeutics (CRSP), 49% surge in interest

Pipeline and key clinical candidates for these companies: 

Cellectis is a clinical-stage biotechnology company that utilizes an allogeneic approach for CAR-T immunotherapies in oncology, “pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients,” and a platform to make therapeutic gene editing in hemopoietic stem cells for various diseases. Cellectis is developing product candidates utilizing TALEN, its gene editing technology, and PulseAgile, its electroporation system to “harness the power of the immune system in order to treat diseases with unmet medical needs,” 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.

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.

PDS Biotech is a clinical-stage immunotherapy company developing a pipeline of targeted cancer and infectious disease immunotherapies based on its proprietary Versamune and Infectimune T cell-activating technology platforms. To date, its lead Versamune clinical candidate, PDS0101, has demonstrated the potential to reduce tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV-positive cancers in multiple Phase 2 clinical trials.

Syndax Pharmaceuticals is a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Highlights of the company’s pipeline include revumenib (SNDX-5613), a highly selective inhibitor of the Menin-MLL binding interaction, and axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor, both currently in pivotal trials.

Axsome Therapeutics is developing and delivering novel therapies for central nervous system, or CNS, conditions that have limited treatment options. AXS-05 is a novel, oral, patent protected, investigational N-methyl-D-aspartate receptor antagonist with multimodal activity under development for the treatment of Alzheimer’s disease agitation and other central nervous system disorders.

Cellectar Biosciences is a late-stage clinical biopharmaceutical company focused on the discovery and development of proprietary drugs for the treatment of cancer, independently and through research and development collaborations. The company’s core objective is to leverage its proprietary Phospholipid Drug Conjugate delivery platform to develop the next-generation of cancer cell-targeting treatments, delivering improved efficacy and better safety as a result of fewer off-target effects. The company’s product pipeline includes lead asset iopofosine, a small-molecule PDC designed to provide targeted delivery of iodine-131, proprietary preclinical PDC chemotherapeutic programs and multiple partnered PDC assets.

Novavax says it promotes improved health by discovering, developing and commercializing innovative vaccines to protect against serious infectious diseases. Novavax offers a differentiated vaccine platform that combines a recombinant protein approach, innovative nanoparticle technology and Novavax’s patented Matrix-M adjuvant to enhance the immune response.

Intellia Therapeutics, a clinical-stage genome editing company, is developing novel, potentially curative therapeutics using CRISPR/Cas9 technology. “Intellia’s deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of CRISPR/Cas9 to create new classes of genetic medicine,” the company says.

CRISPR Therapeutics is a gene editing company focused on developing gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. “CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte,” the company states.

Recent news on these stocks:

October 3

RedHill Biopharma announced that novel, twice daily, oral opaganib delivered a statistically significant increase in survival time when given at 150 mg/kg twice a day in a U.S. Army Medical Research Institute of Infectious Diseases in vivo Ebola virus study, making it the first host-directed molecule to show activity in Ebola virus disease. The U.S. Army study tested three doses of opaganib against an inactive vehicle control arm. The in vivo study results showed a statistically significant survival increase in mean survival time of 11.2 days in the 150 mg/kg opaganib group compared to a mean survival time of 5.5 days in the inactive vehicle control group. A 30% mice survival was observed in the 150 mg/kg treated group compared to the vehicle control. Twice daily administered opaganib has previously demonstrated antiviral benefit in late-stage clinical studies of patients hospitalized with moderate to severe COVID-19 and was selected by the NIH Radiation and Nuclear Countermeasures Program for Acute Radiation Syndrome development.

PDS Biotechnology announced updated interim data based on an August 2nd cut off from the VERSATILE-002 Phase 2 clinical trial evaluating PDS0101 in combination with Merck’s anti-PD-1 therapy, KEYTRUDA, in patients with unresectable, recurrent, or metastatic HPV16-positive head and neck squamous cell carcinoma, HNSCC. VERSATILE-002 is investigating two patient populations whose cancer has returned or spread – ICI naive and ICI refractory. The ICI naive group had not responded to standard-of-care treatments but had not yet been treated with an ICI. The ICI refractory group included patients who had not responded to multiple prior treatments, including ICI therapy. Data presented at ASCO was based on a January 13th cut off. VERSATILE-002: ICI Naive. Highlights of the interim data from the ICI naive cohort include: 24-month overall survival rate is 74%; published 24-month survival rate of less than 30% for approved ICI; 12-month OS rate is 80%; published results of 30-50% with approved ICIs; Tumor shrinkage seen in 60% of patients; Confirmed overall response rate is 27% to date; Median progression-free survival is 8.1 months to date; published results of 2-3 months PFS with approved ICIs; 13% of patients experienced Grade 3 treatment-related adverse events and 0% experienced Grade 4 or 5 TRAE; published results report 13-17% Grade 3-5 TRAE with approved ICI monotherapy; 60% of patients have CPS score of 1-19, and 40% have CPS score greater than20. VERSATILE-002: ICI Refractory: The goal of this ICI refractory cohort was to confirm and to better understand the role of PDS0101 in prolonging the survival of advanced HPV16-positive head and neck cancer patients who received PDS0101 in combination with KEYTRUDA. This analysis is also intended to provide insight to the contribution of PDS0101 to overall survival in the National Cancer Institute-led study evaluating the combination of PDS0101, PDS0301, and an ICI. Highlights of the interim data from the ICI refractory cohort include: The 12-month OS rate is 56%. The published median 12-month OS rate is 17% with no salvage chemotherapy following tumor progression on ICI; 0% confirmed ORR suggests that PDS0101’s impact on survival does not appear to be dependent on tumor shrinkage; 4% of patients experienced Grade 3 TRAE and 0% patients experienced Grade 4 and 5 TRAE.

Novavax announced that the Novavax COVID-19 Vaccine, Adjuvanted has received Emergency Use Authorization from the U.S. Food and Drug Administration for active immunization to prevent COVID-19 in individuals aged 12 and older. Immediately upon authorization, Novavax’s vaccine has also been included in the recommendations issued by the U.S. Centers for Disease Control and Prevention on September 12, 2023. Doses will be available nationwide at thousands of locations, including national and local retail pharmacies and physicians’ offices, following the Center for Biologics Evaluation and Research release of vaccine batches, expected in the coming days. “COVID-19 is once again on the rise with infections and hospitalizations increasing, so it’s important that individuals get vaccinated to protect themselves and their loved ones,” said John C. Jacobs, President and Chief Executive Officer, Novavax. “Novavax’s authorization today means people will now have the choice of a protein-based non-MRNA option to help protect themselves against COVID-19, which is now the fourth leading cause of death in the U.S. In the coming days, individuals in the U.S. can go to pharmacies, physicians’ offices, clinics and various government entities to receive an updated Novavax vaccine.”

Regeneron Pharmaceuticals (REGN) and Intellia Therapeutics announced an expanded research collaboration to develop additional in vivo CRISPR-based gene editing therapies focused on neurological and muscular diseases. This builds on the success of the companies’ existing collaboration and continues to combine both companies’ deep biology and technology expertise. The collaboration will leverage Regeneron’s proprietary antibody-targeted adeno-associated virus vectors and delivery systems and Intellia’s proprietary Nme2 CRISPR/Cas9 systems adapted for viral vector delivery and designed to precisely modify a target gene. Under the terms of the expanded agreement, the companies will initially research two in vivo non-liver targets. Intellia will lead the design of the editing methodology and Regeneron will lead the design of the targeted viral vector delivery approach. Each company will have the opportunity to lead potential development and commercialization of product candidates for one target, and the company that is not leading development and commercialization will have the option to enter into a co-development and co-commercialization agreement for the target.

October 2

Syndax Pharmaceuticals announced positive topline data from the protocol-defined pooled analysis of the pivotal AUGMENT-101 trial of revumenib, a first-in-class menin inhibitor, in adult and pediatric patients with relapsed/refractory KMT2A-rearranged acute myeloid leukemia and acute lymphoid leukemia. The AUGMENT-101 trial met its primary endpoint at the protocol-defined interim analysis stage with a complete remission or a CR with partial hematological recovery rate of 23% among the 57 efficacy evaluable patients in the pooled KMT2Ar acute leukemia cohort. The CR/CRh rate in patients with KMT2Ar AML was 24.5%. The CR/CRh responses in both the overall population and the AML subset were durable with a 6.4-month median duration as of the July 24, 2023 data cut-off, with 46% remaining in response. Minimal residual disease status was assessed in 10 of the 13 patients who achieved a CR/CRh, 70% of whom were MRD negative. In the efficacy-evaluable patients, the overall response rate1 was 63%. A total of 14 patients who achieved an overall response underwent hematopoietic stem cell transplant, eight of whom did not achieve a CR or CRh prior to transplant. Half of the patients who had an HSCT received post-transplant maintenance with revumenib and three additional patients were in follow-up and are eligible to restart revumenib as post-transplant maintenance. Based on the Independent Data Monitoring Committee recommendation, the company is stopping the trial to further accrual in the KMT2Ar cohorts. Syndax continues to expect to submit an NDA for revumenib for the treatment of R/R KMT2Ar acute leukemia to the U.S. FDA by year-end.

Cellectar Biosciences announced significant advancements to its global intellectual property portfolio. Cellectar has secured four patents across the key global regions Europe, Australia and Canada, covering the company’s proprietary Phospholipid Drug Conjugate delivery platform and lead drug in development, iopofosine I 131. The European Patent Office has granted patent application number 20172745.0, titled “Ether Phospholipid Compounds for Treating Cancer and Imaging Detection of Cancer Stem Cells”. This patent significantly enhances the use of iopofosine I 131 for treating various cancers, including gliomas, lung cancer, melanoma, and more, with a focus on cancer stem cells. It also extends coverage for iopofosine I 131’s application in underserved pediatric diseases, supporting upcoming clinical advancements. The Canadian Intellectual Property Office, the Australian Patent Office and the EPO have granted patent number 17814042.2 for “Phospholipid Ether Analogs for the Identification and Isolation of Circulating Tumor Cells”. This patent encompasses a method using cancer-targeted alkylphosphocholine analogs to effectively identify and isolate circulating tumor cells from various cancer types, providing a valuable diagnostic and research tool in the field of oncology. It underscores Cellectar’s ability to target not only primary tumors but also elusive tumor cells in challenging locations, such as the bloodstream, and metastatic tissue which are often seeded by circulating tumor cells marking a significant advancement in developing targeted cancer treatments. The IP Australia and the EPO have granted patent application number WO 2019199998 A1, titled “Fractionated Dosing Of A Phospholipid Ether Analog For The Treatment Of Cancer”. This patent represents a significant milestone for Cellectar, extending comprehensive protection to the company’s innovative fractionated dosing regimen of iopofosine I 131 for the targeted treatment of cancer. The EPO has granted patent number EP3229810 (B1) titled “Phospholipid Ether Analogs as Cancer-Targeting Drug Vehicles”, which extends protection to Cellectar Biosciences’ phospholipid ether (PLE) analogs for precisely and selectively delivering radiation to tumors and cancer stem cells, thus minimizing radiation exposure to normal tissues. This patent encompasses both the composition of matter and the method of use for the company’s PDC(TM) products, including iopofosine I 131, and enables the development of targeted anticancer agents for various hematologic and solid tumors.

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.