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: 

• Exicure (XCUR), 1,935% surge in interest
• Aclaris Therapeutics (ACRS), 1,788% surge in interest
• Fate Therapeutics (FATE), 794% surge in interest
• Hoth Therapeutics (HOTH), 467% surge in interest
• Regenxbio (RGNX), 268% surge in interest
• Incyte (INCY), 237% surge in interest
• Apellis Pharmaceuticals (APLS), 162% surge in interest
• Protalix Biotherapeutics (PLX), 62% surge in interest
• Tonix Pharmaceuticals (TNXP), 127% surge in interest

Pipeline and key clinical candidates for these companies:

Exicure is an early-stage biotechnology company historically focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets. The company says it continues to actively pursue out-licensing opportunities for its clinical asset, cavrotolimod, as well as for its preclinical candidates, including the SCN9A program for neuropathic pain, and to pursue all strategic alternatives with the goal of maximizing stockholder value.

Aclaris Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates to address the needs of patients with immuno-inflammatory diseases who lack satisfactory treatment options. The company has a multi-stage portfolio of drug candidates powered by a robust R&D engine exploring protein kinase regulation.

Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell-derived cellular immunotherapies to patients with cancer and autoimmune disorders. Using its proprietary iPSC product platform, “the company has established a leadership position in creating multiplexed-engineered iPSC lines and in the manufacture and clinical development of off-the-shelf, iPSC-derived cell products,” the company states.

Hoth Therapeutics refers to itself as “a catalyst in early-stage pharmaceutical research and development, elevating drugs from the bench to pre-clinical and clinical testing.” Hoth “collaborates and partners with a team of scientists, clinicians, and key opinion leaders to seek out and investigate therapeutics that hold immense potential to create breakthroughs and diversify treatment options,” the company stated.

Regenxbio is a clinical-stage biotechnology company that says it is “seeking to improve lives through the curative potential of gene therapy.” Regenxbio’s NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8 and AAV9, the company has noted.

Incyte is a global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics.

Apellis Pharmaceuticals says the company “ushered in the first new class of complement medicine in 15 years” with the approval of the first and only targeted C3 therapy. Apellis is advancing this science to “continually develop transformative medicines for people living with rare, retinal, and neurological diseases,” it stated.

Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. It is the first company to gain FDA approval of a protein produced through plant cell-based in suspension expression system. This unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights to taliglucerase alfa for the treatment of Gaucher disease, Protalix’s first product manufactured through ProCellEx, excluding in Brazil, where Protalix retains full rights. Protalix’s second product, Elfabrio, was approved by both the FDA and the European Medicines Agency in May 2023.

Tonix is focused on central nervous system, rare disease, immunology and infectious disease product candidates. Tonix’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia.

Recent news on these stocks:

November 20

Hoth Therapeutics announced that the company’s board of directors approved the purchase of up to $1M in Bitcoin as a treasury reserve asset.

November 19

Astellas Pharma (ALPMY) announced the FDA issued a Complete Response Letter regarding the supplemental new drug application for Izervay for the treatment of geographic atrophy secondary to age-related macular degeneration. The drug application sought to include positive two-year data in the U.S. Prescribing Information for Izervay based on results from the GATHER2 Phase 3 clinical trial, which evaluated the efficacy and safety of monthly and every other month dosing through year two. The FDA stated the agency cannot approve the application in its present form by the action date of November 19. “The FDA comments outlined in the CRL are unrelated to the safety and benefit/risk of the use of IZERVAY; rather, the comments focus on a statistical matter related to labelling language proposed by Astellas. Astellas is seeking further clarification from the FDA and looks forward to working with the agency to quickly address the agency’s feedback,” the company said in a statement. Shares of Apellis, whose Syfovre competes with Izervay, rose 3% to $26.99 in premarket trading.

November 18

Aclaris Therapeutics has entered into an exclusive license agreement with Biosion for worldwide rights to BSI-045B, a potential best-in-class, clinical-stage, novel anti-TSLP monoclonal antibody, and BSI-502, a potential best-in-class, pre-clinical stage, novel bispecific antibody that is directed against both TSLP and IL4R. In a completed Phase 2a, single-arm, proof-of-concept trial in the United States in 22 patients with moderate to severe atopic dermatitis, BSI-045B was observed to have a pharmacodynamic, safety and efficacy profile that could position it as a potential best-in-class therapy. BSI-045B is also being advanced in multiple Phase 2 studies in China by Biosion’s regional partner, Chia Tai Tianqing Pharmaceutical Group, targeting both severe asthma and chronic rhinosinusitis with nasal polyps, accelerating the potential to show proof-of-concept across additional indications. Biosion is a global biotechnology company that has built a pipeline of innovative biologics through its internally derived proprietary technologies. Concurrent with the Biosion transaction, Aclaris has made two key additions to its leadership team: Hugh Davis, Ph.D., joins as President and COO, bringing over 35 years of experience in biologics development, clinical pharmacology, and business development from leadership roles at Biosion, Frontage, GSK, and Johnson & Johnson. Steven Knapp, PharmD, joins as Executive Vice President, Head of Regulatory & Quality, with over 35 years of regulatory and quality experience from senior positions at Biosion, Antares, Valeant, and BMS.

Fate Therapeutics presented initial clinical and translational data from the company’s Phase 1 study of FT522 in relapsed / refractory B-cell lymphoma at the American College of Rheumatology Convergence in Washington, D.C. FT522 is the company’s off-the-shelf, CD19-targeted chimeric antigen receptor – CAR – natural killer – NK – cell product candidate that incorporates multiple novel synthetic controls of cell function designed to target and deplete pathogenic immune cells, and is the company’s first product candidate to integrate its alloimmune defense receptor technology to enable effective treatment of patients without administration of intense conditioning chemotherapy. The Phase 1 basket study for B cell-mediated autoimmune diseases is designed to assess FT522 as add-on to standard-of-care induction and maintenance regimens without conditioning chemotherapy. Initial Phase 1 clinical data in relapsed / refractory B-cell lymphoma show favorable safety profile, complete responses, and persistence of FT522 live cells. Selective targeting and reduction of CD19+ B cells was observed with each FT522 dose in the study’s first low-dose cohort without conditioning chemotherapy, supporting novel ADR technology.

Regenxbio announced that AFFINITY DUCHENNE, the multi-center, open-label trial of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy, has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety data from the Phase I/II portion of the study, including the first functional data. Affinity Duchenne data updates: Results include 12-month data from three dose level 1 patients aged 4-10 and nine-month data from two dose level 2 patients aged 8 and 12. RGX-202 was well tolerated with no serious adverse events and no AEs of special interest. Phase I/II data show RGX-202 recipients exceeding external natural history and established benchmarks for clinical outcomes: Functional improvements seen in all patients treated with dose level 1 and dose level 2 at 12 and 9 months respectively; New biomarker data confirms consistent robust expression of differentiated RGX-202 microdystrophin in the muscle; Favorable safety profile observed at both dose levels; no serious adverse events or AEs of special interest

Incyte announced that it will pause enrollment in the ongoing Phase 2 study of MRGPRX2 in chronic spontaneous urticaria, CSU. The decision was made following the observation of certain in vivo preclinical toxicology findings. These data have been shared with the FDA and Incyte will work closely with the FDA to determine next steps. Enrollment in the other INCB000262 proof-of-concept studies is complete and data from all clinical studies will help inform its future development and guide the potential development of back-up molecules. In addition, data from the Phase 2 study evaluating MRGPRX4 in cholestatic pruritus, CP, does not support further development.

Tonix Pharmaceuticals presented data in a poster presentation at the ACR Convergence 2024 Annual Meeting, held November 14-19, 2024, in Washington, D.C. A copy of the Company’s presentation, titled “Randomized, Double-Blind, Placebo-Controlled Confirmatory Phase 3 Trial of Bedtime Sublingual Cyclobenzaprine in Fibromyalgia” is available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com. In the Phase 3 RESILIENT study, TNX-102 SL met the pre-specified primary endpoint of significantly reducing daily pain compared to placebo in participants with fibromyalgia. In the RESILIENT study, TNX-102 SL demonstrated a broad spectrum of benefits with statistically significant improvement in all six pre-specified key secondary endpoints including those related to improved sleep quality, reduced fatigue, and improved patient global ratings and overall fibromyalgia symptoms and function. TNX-102 SL was generally well tolerated with an adverse event profile comparable to prior studies and no new safety signals observed. Tonix submitted a new drug application to the U.S. Food and Drug Administration in October 2024 for TNX-102 SL for the management of fibromyalgia. The FDA typically has a 60-day filing review period to determine whether the submitted NDA is complete and accepted for review. If the FDA accepts the NDA for review, the Company expects a 2025 date for a FDA decision on approval, based on the Prescription Drug User Fee Act.

November 14

Exicure entered into two share purchase agreements, stating,”Exicure has historically been an early-stage biotechnology company focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets. In September 2022, the company announced a significant reduction in force, suspension of preclinical activities and halting of all research and development, and that the company was exploring strategic alternatives to maximize stockholder value. In an agreement dated November 6, and executed on November 12, the company agreed to sell and issue to HiTron Systems 433K shares of common stock for $1.3M, at a purchase price of $3.00 per share. The closing of this transaction is expected to occur within 10 days from its execution. On November 13, in a subsequent agreement, the company agreed to sell and issue to HiTron 2.9M additional shares of common stock for $8.7M, at a purchase price of $3.00 per share. The closing of the investment pursuant to this subsequent agreement is conditioned on stockholder approval, among other customary conditions.”

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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.