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), 9,100% surge in interest
• Aldeyra (ALDX), 2,187% surge in interest
• Cohbar (CWBR), 1,657% surge in interest
• Ocular Therapeutics (OCUL), 900% surge in interest
• TG Therapeutics (TGTX), 826% surge in interest
• Sarepta (SRPT), 734% surge in interest
• Plus Therapeutics (PSTV), 689% surge in interest
• CASI Pharmaceuticals (CASI), 449% surge in interest
• Repligen (RGEN), 259% surge in interest
• X4 Pharmaceuticals (XFOR), 247% 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.

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.

CohBar is a clinical-stage biotechnology company leveraging the power of the mitochondria and the peptides encoded in its genome to develop potential breakthrough therapeutics targeting chronic and age-related diseases with limited to no treatment options.

Ocular Therapeutix is focused on the formulation, development, and commercialization of therapies for diseases and conditions of the eye using its proprietary bioresorbable hydrogel-based formulation technology. Ocular Therapeutix’s first commercial drug product, Dextenza, is an FDA-approved corticosteroid for the treatment of ocular inflammation and pain following ophthalmic surgery and ocular itching associated with allergic conjunctivitis. Ocular Therapeutix’s earlier stage development assets include: OTX-TKI, currently in Phase 1 clinical trials for the treatment of wet AMD and diabetic retinopathy; OTX-TIC, currently in a Phase 2 clinical trial for the treatment of primary open-angle glaucoma or ocular hypertension; and OTX-CSI for the chronic treatment of dry eye disease and OTX-DED for the short-term treatment of the signs and symptoms of dry eye disease, both of which have completed Phase 2 clinical trials.

TG Therapeutics is focused on the acquisition, development and commercialization of novel treatments for B-cell diseases. In addition to a research pipeline including several investigational medicines, TG has received approval from the FDA for Briumvi, for the treatment of adult patients with relapsing forms of multiple sclerosis, to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease.

Sarepta Therapeutics engineers precision genetic medicine for rare diseases. The company holds leadership positions in Duchenne muscular dystrophy, or DMD, and limb-girdle muscular dystrophies, or LGMDs, and currently has more than 40 programs in various stages of development. Sarepta’s pipeline is driven by its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.

Plus Therapeutics is a clinical-stage pharmaceutical company developing targeted radiotherapeutics for difficult-to-treat cancers of the central nervous system with the potential to enhance clinical outcomes for patients. Combining image-guided local beta radiation and targeted drug delivery approaches, the Company is advancing a pipeline of product candidates with lead programs in recurrent glioblastoma and leptomeningeal metastases.

CASI Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products in China, the United States, and throughout the world. The company is focused on acquiring, developing, and commercializing products that augment its hematology oncology therapeutic focus as well as other areas of unmet medical need. The company intends to execute its plan to become a leader by launching medicines in the greater China market, leveraging the company’s China-based regulatory and commercial competencies and its global drug development expertise.

Repligen Corporation develops and commercializes bioprocessing technologies and systems that enable efficiencies in the process of manufacturing biological drugs. The customers served are primarily biopharmaceutical drug developers and contract development and manufacturing organizations, or CDMOs.

X4 Pharmaceuticals is focused on the discovery and development of novel therapies for people with diseases of the immune system. The company’s lead clinical candidate is mavorixafor, a small molecule antagonist of chemokine receptor CXCR4 that is being developed as an oral, once-daily therapy.

Recent news on these stocks:

November 2

Repligen announced the commercial launch of TangenX SC, the industry’s first holder-free, self-contained Tangential Flow Filtration, TFF, device. The technology is especially suited to the manufacture of biologics where fully closed systems are critical or ideal, including antibody drug conjugates, viral vectors, nucleic acids and lipid nanoparticles, and can also be applied to monoclonal antibody, and recombinant protein production. Christine Gebski, Senior Vice President, Filtration and Chromatography at Repligen, said, “TangenX SC pushes the boundaries of TFF innovation, offering clear advantages to virtually any user of flat sheet TFF. The holder-free TangenX SC device simplifies and streamlines downstream flat sheet UF/DF processes like never before. Users can trust that the quality and consistency of their TFF processes remain uncompromised while enjoying the benefits of this innovative technology.”

November 1

AstraZeneca (AZN) announced a collaboration and investment agreement with Cellectis, a clinical-stage biotechnology company, to accelerate the development of next generation therapeutics in areas of high unmet need, including oncology, immunology and rare diseases. Under the terms of the collaboration agreement, AstraZeneca will leverage the Cellectis proprietary gene editing technologies and manufacturing capabilities, to design novel cell and gene therapy products, strengthening AstraZeneca’s growing offering in this space. As part of the agreement, 25 genetic targets have been exclusively reserved for AstraZeneca, from which up to 10 candidate products could be explored for development. In Q4 2023, Cellectis will receive an initial payment of $105M from AstraZeneca, which comprises a $25M upfront cash payment under the terms of a research collaboration agreement and an $80M equity investment. The initial equity investment of $80M, at $5/share, represents an equity stake of c. 22% in Cellectis. A further $140M equity investment, at $5/share, is expected to close in early 2024 subject to the signing of a final binding agreement following completion of a consultation with the Cellectis employee representative bodies and customary closing conditions including Cellectis shareholders’ approval and regulatory clearances. Post-closing of this second investment, AstraZeneca will hold a total equity stake of c. 44% in Cellectis. AstraZeneca expects to treat its investment in Cellectis as an associate. Under the terms of the research collaboration, Cellectis is also eligible to receive an investigational new drug, or IND, option fee and development, regulatory and sales-related milestone payments, ranging from $70M up to $220M, per each of the 10 candidate products, plus tiered royalties. AstraZeneca retains an option for a worldwide exclusive license for the candidate products developed under the research collaboration agreement, to be exercised before IND filing. Marc Dunoyer, Chief Strategy Officer, AstraZeneca, and Chief Executive Officer, Alexion, AstraZeneca Rare Disease, said: “The differentiated capabilities Cellectis has in gene editing and manufacturing complement our in-house expertise and investments made in the past year. AstraZeneca continues to advance our ambition in cell therapy for oncology and autoimmune diseases as well as in genomic medicine, which has potential to be transformative for patients with rare diseases.”

Aldeyra Therapeutic (ALDX) announced that it has entered into an exclusive option agreement with AbbVie (ABBV). Under the terms of the option agreement, AbbVie has the option to acquire a co-exclusive license to develop, manufacture, and commercialize reproxalap in the U.S. and an exclusive license to develop, manufacture, and commercialize reproxalap outside the U.S. Aldeyra will receive a non-refundable option fee of $1M and an upfront payment of $100M less option fees if AbbVie chooses to exercise the option. Under the terms of the license agreement, Aldeyra would be eligible to receive up to $300M in regulatory and commercial milestone payments, inclusive of a $100M milestone payment upon U.S. Food and Drug Administration approval of reproxalap in dry eye disease; in the United States, Aldeyra and AbbVie would share profits and losses from the commercialization of reproxalap according to a split of 60% for AbbVie and 40% for Aldeyra; and for markets outside the U.S., Aldeyra would be eligible to receive tiered royalties on net sales of reproxalap. Exercise of the option will also grant AbbVie the right of first negotiation for compounds that are owned or otherwise controlled by Aldeyra in the field of ophthalmology relating to treating conditions of the ocular surface. The right of first negotiation is in addition to a right to review data for any other compounds that are owned or otherwise controlled by Aldeyra in the fields of ophthalmology and immunology before such data is shared with any other third party. Additional details regarding the terms of the option agreement may be found in a Current Report on Form 8-K filed by Aldeyra with the Securities and Exchange Commission.

Ocular Therapeutix announced it had received written agreement regarding the overall design from the U.S. Food and Drug Administration, FDA, under a Special Protocol Assessment, SPA, for the Company’s ongoing pivotal Phase 3 clinical trial for AXPAXLI, for the treatment of wet age-related macular degeneration, which the Company refers to as the SOL trial. The Company initiated the SOL trial in September 2023 and expects to enroll approximately 300 evaluable wet AMD subjects who are treatment naive in the study eye in the trial. The SOL trial is designed to be a multi-center, parallel-group trial run primarily at U.S. sites, with subjects randomized to one injection of aflibercept or one implant of AXPAXLI followed by supplemental anti-VEGF treatment based on pre-specified criteria. With the agreement under the SPA, the Company will begin enrolling patients in the SOL trial and expects to dose the first subject by year-end.

TG Therapeutics reported Q3 EPS of 73c, consensus 12c, and Q3 revenue of $165.8M, consensus $46.56M. CEO Michael Weiss stated, “The team has executed very well in the third quarter, making significant progress on our corporate goals and continuing to build a solid foundation for the BRIUMVI launch. We showed strong net quarterly revenue of approximately $166 million, including an upfront milestone payment from our ex-U.S. partner, Neuraxpharm, as well as $25.1 million in BRIUMVI net sales in the U.S., which again exceeded our expectations.” Mr. Weiss continued, “The adoption of BRIUMVI from both healthcare providers and centers continues to grow, which I believe positions us to close out the year on a positive note and I am excited for 2024 and for the future of BRIUMVI and TG.”

October 31

Plus Therapeutics reported Q3 EPS of ($1.00), consensus ($1.12), and Q3 revenue of $1.24M, consensus $1.29M. Additionally, Plus Therapeutics announced that its board approved a share repurchase program, with authorization to repurchase up to $500K of the company’s outstanding common stock.

X4 Pharmaceuticals announced that the FDA has accepted for filing the company’s New Drug Application, NDA, for once-daily, oral mavorixafor to treat individuals aged 12 and older with WHIM syndrome, a rare, primary immunodeficiency. The FDA granted Priority Review of the mavorixafor NDA, establishing a goal of six months review from the date of acceptance and assigning a Prescription Drug User Fee Act, PDUFA, target action date of April 30, 2024. “The FDA’s acceptance of our mavorixafor NDA with priority review represents yet another significant step forward towards a potential treatment for those with WHIM syndrome, a rare disease for which there are currently no approved therapies,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “We look forward to working closely with the FDA throughout the NDA review process with the goal of bringing mavorixafor to people with WHIM syndrome as quickly as possible.”

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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.