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: 

• CNS Pharmaceuticals (CNSP), 464% surge in interest
• Bio-Path Holdings (BPTH), 328% surge in interest
• Ocular Therapeutix (OCUL), 258% surge in interest
• Vaxart (VXRT), 101% surge in interest
• UroGen Pharmaceuticals (URGN), 69% surge in interest
• Cabaletta Bio (CABA), 65% surge in interest

Pipeline and key clinical candidates for these companies:

CNS Pharmaceuticals is a clinical-stage pharmaceutical company developing a pipeline of anti-cancer drug candidates for the treatment of primary and metastatic cancers of the brain and central nervous system. The company’s lead drug candidate, Berubicin, is a novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier. Berubicin is currently in development for the treatment of a number of serious brain and CNS oncology indications including glioblastoma multiforme, or GBM, an aggressive and incurable form of brain cancer.

Bio-Path is developing DNAbilize, a technology that has yielded a pipeline of RNAi nanoparticle drugs that can be administered with an intravenous transfusion. Bio-Path’s lead product candidate, prexigebersen , or BP1001, is in a Phase 2 study for blood cancers and BP1001-A, a drug product modification of prexigebersen, has been cleared by the FDA and Phase 1 studies in solid tumors will commence in 2022. The company’s second product, BP1002, is being evaluated for the treatment of blood cancers and solid tumors, including lymphoma and acute myeloid leukemia. In addition, an IND is expected to be filed for BP1003, a novel liposome-incorporated STAT3 antisense oligodeoxynucleotide developed by Bio-Path as a specific inhibitor of STAT3, in 2022 or Q1 of 2023.

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.

Vaxart is a clinical-stage biotechnology company developing a range of oral recombinant vaccines based on its proprietary delivery platform. Vaxart believes that its proprietary pill vaccine delivery platform is “suitable to deliver recombinant vaccines, positioning the company to develop oral versions of currently marketed vaccines and to design recombinant vaccines for new indications,” the company has stated. Vaxart’s development programs currently include pill vaccines designed to protect against norovirus, coronavirus, seasonal influenza, and respiratory syncytial virus, or RSV, as well as a therapeutic vaccine for human papillomavirus, or HPV, Vaxart’s first immune-oncology indication.

UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers. UroGen has developed RTGel reverse-thermal hydrogel, a proprietary sustained-release, hydrogel-based platform technology that it says has the potential to improve the therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. The company’s first product to treat low-grade upper tract urothelial cancer and investigational treatment UGN-102 for intravesical solution for patients with LG-IR-NMIBC are designed to ablate tumors by non-surgical means.

Caballeta Bio is focused on the discovery and development of engineered T cell therapies that have the potential to provide a deep and durable, perhaps curative, treatment for patients with autoimmune diseases. The CABA platform encompasses chimeric antigen receptor T cells for autoimmunity and Cabaletta Bio’s proprietary chimeric autoantibody receptor T cells.

Recent news on these stocks:

June 14

CNS Pharmaceuticals entered into securities purchase agreements with institutional investors for the sale by the company of 336K shares of the company’s common stock and pre-funded warrants to purchase 30K shares of common stock in lieu thereof in a registered direct offering. In a concurrent private placement , the company also sold to the investors unregistered warrants to purchase up to an aggregate of 366K shares of common stock. The combined purchase price of one share of common stock and accompanying common warrant is $3.75. The closing of the offering and private placement is subject to customary closing conditions and is expected to occur on June 17.

Bio-Path Holdings presented interim results from the company’s Phase 2 study of prexigebersen in combination with decitabine and venetoclax for the treatment of acute myeloid leukemia in a poster presentation at 2024 European Hematology Association Congress, on June 14, 2024 in Madrid, Spain. Jorge Cortes, M.D., Director of the Georgia Cancer Center, presented data showing prexigebersen continues to be well-tolerated and has now demonstrated compelling efficacy results in two reporting cohorts including evaluable newly diagnosed AML patients and evaluable refractory/relapsed AML patients, both of which exceeded outcomes with frontline therapy. In Cohort 1, 31 newly diagnosed patients were enrolled; 20 evaluable patients with a median age of 75 years, treated with at least one cycle of prexigebersen, decitabine and venetoclax, had adverse-risk or secondary AML evolved from myelodysplastic syndromes, chronic myelomonocytic leukemia or treatment-related AML. Fifteen patients achieved complete remission, CRh, or CRi; two patients achieved partial remission and two patients achieved stable disease. In Cohort 2, 38 relapsed/refractory patients were enrolled; 23 evaluable patients with a median age of 63 years, treated with at least one cycle of prexigebersen, decitabine and venetoclax, had adverse-risk or sAML. Twelve patients achieved CR/CRi/CRh; one patient achieved PR, eight patients achieved SD and one patient had treatment failure. Among the evaluable patients of both cohorts, adverse events were consistent with those expected with decitabine and venetoclax and/or AML, including fatigue, anemia and neutropenia, while the most frequent severe adverse events were febrile neutropenia and sepsis. Given these promising interim results, Bio-Path expects to continue enrollment of up to 98 and 54 evaluable patients for Cohorts 1 and 2, respectively.

Cabaletta Bio reported initial clinical data from each of the first two patients dosed with CABA-201 in the Phase 1/2 RESET-Myositis and RESET-SLE trials. These data will be presented at a EULAR European Congress of Rheumatology 2024 Industry Symposia session titled “Immune Reset: The Potential of CAR T Cell Therapy to Transform the Treatment of Patients with Autoimmune Disease” in Vienna, Austria. Cabaletta designed CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, to deeply and transiently deplete CD19-positive B cells following a one-time infusion that may enable a reset of the immune system with the potential for durable remission without chronic therapy in patients with autoimmune diseases. Cabaletta is advancing four Phase 1/2 RESET trials evaluating CABA-201 within a total of ten cohorts with six patients in each cohort. All cohorts are evaluating the same single, weight-based dose of 1 x 106 cells/kg, following a preconditioning regimen of fludarabine and cyclophosphamide consistent with the dosing regimen used in the academic experience, without a dose escalation requirement. As of May 28, the data cut-off date, one patient treated in the immune-mediated necrotizing myopathy cohort in the RESET-Myositis trial had completed three months of follow-up and one patient enrolled in the systemic lupus erythematosus non-renal cohort in the RESET-SLE trial had completed one month of follow-up. The patient with IMNM is a 33-year-old male with a two-year history of disease, positive for anti-SRP antibody and who had prior disease-specific therapy that included IVIg, rituximab, methotrexate and glucocorticoids. The patient with SLE is a 26-year-old male with a six-year history of disease, positive for anti-dsDNA antibody and who had prior disease specific therapy that included cyclophosphamide, voclosporin, belimumab, tacrolimus, mycophenolate mofetil, hydroxychloroquine and glucocorticoids. Both patients were administered a one-time infusion of CABA-201 at 1 x 106 cells/kg, following a preconditioning regimen of fludarabine and cyclophosphamide. The primary endpoint of each trial is safety and tolerability within 28 days of infusion. Secondary endpoints include translational assessments and clinical outcomes. CABA-201 was administered during a four-day hospital stay, as currently required by the protocol, and was generally well-tolerated with no serious adverse events reported for either patient through the follow-up period. No evidence of cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome of any grade was observed for either patient through the follow-up period. Tocilizumab was not administered for either patient. No infections were observed for either patient through the follow-up period. All chronic maintenance therapy or concomitant medications were discontinued for both patients through the follow-up period, other than a planned prednisone taper for the SLE patient. Complete B cell depletion was observed within 15 days post-infusion with CABA-201 in both patients. Both patients had early, transient leukopenia, as expected with the preconditioning regimen. CAR T cell expansion associated with CABA-201 reached its peak magnitude at day 15 post-infusion in both patients and the magnitude of expansion was consistent with the academic experience with a similar 4-1BB CD19-CAR T construct. At week 12 of follow-up for the IMNM patient, the data show a decline in creatinine kinase from 617 at infusion to 308 and a total improvement score of 30, which is consistent with the clinically meaningful improvement seen in the academic experience of a similar 4-1BB CD19-CAR T construct that also recently reported data from an IMNM patient. At week 4 of follow-up for the SLE patient, the data demonstrated an improvement in the SLEDAI-2K score from 26 at baseline to 10. B cell repopulation was observed in the IMNM patient at week 8 with immature, naive B cell phenotypes as demonstrated by flow cytometry, suggesting potential immune system reset with confirmatory analyses ongoing.

June 13

Ocular Therapeutix hosted an Investor Day where it highlighted excellent clinical development progress with AXPAXLI for wet age-related macular degeneration wet AMD and non-proliferative diabetic retinopathy NPDR and updated its corporate strategy. “2024 has been a time of significant accomplishment for Ocular. To support our mission to become a leader in retinal care, we successfully assembled a ‘Retina Dream Team’ of recognized leaders in the field, fortified our balance sheet with additional capital and streamlined the organization. We are already seeing the positive impact of these efforts, based on exceptional enrollment in the pivotal SOL-1 AXPAXLI study for wet AMD, coupled with plans for a new SOL-R repeat dosing study and the report of positive 48-week topline data from the Phase 1 HELIOS NPDR study,” said Pravin U. Dugel, MD, Executive Chairman, President and Chief Executive Officer of Ocular Therapeutix. “Our transformation into a retina-focused company is built on three pillars: convincing data from three clinical studies with AXPAXLI, de-risking the regulatory pathway in wet AMD, and a focus on expansive, retinal vascular disease markets. We are more confident today than ever before that AXPAXLI has demonstrated monotherapy activity, with potential best-in-class durability and a favorable safety profile that is well positioned to disrupt today’s treatment paradigm which includes frequent, burdensome regimens.”

Vaxart announced that it received a project award valued at up to $453M through the Rapid Response Partnership Vehicle, or RRPV. The RRPV is a Consortium funded by the Biomedical Advanced Research and Development Authority, or BARDA, part of the Administration for Strategic Preparedness and Response, or ASPR, in the U.S. Department of Health and Human Services, or HHS. The funds will be used to conduct a Phase 2b comparative study evaluating Vaxart’s oral pill COVID-19 vaccine candidate against a FDA-approved mRNA vaccine comparator. In preparation for the trial, Vaxart created and manufactured under Good Manufacturing Practice, or GMP, standards a next-generation oral COVID-19 vaccine tablet candidate that – based on preclinical data – is more potent than Vaxart’s prior COVID-19 vaccine constructs. Funding under the award will be provided in two parts with approximately $65.7M available immediately to continue study start-up activities, and the remainder of approximately $387.2M provided when Vaxart and BARDA have determined that the study may further proceed and paid over the course of the study. Currently, Vaxart anticipates initiating enrollment as early as summer 2024. An interim analysis for vaccine efficacy compared to an approved mRNA comparator may occur as early as the first quarter of 2025.

UroGen Pharma announced 82.3% 12-month duration of response data by Kaplan-Meier estimate from its Phase 3 ENVISION trial in patients who achieved complete response at three months after the first instillation of investigational drug UGN-102 for intravesical solution. The ENVISION trial previously met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.6% CR rate at three months following the first instillation of UGN-102. The ENVISION Phase 3 study is investigating UGN-102 in patients with low-grade intermediate-risk non-muscle invasive bladder cancer. In addition to the 12-month data, the DOR Kaplan Meier estimates at 15 and 18 months were both 80.9%. “UGN-102 has demonstrated a strong clinical profile across multiple trials, with these latest results of 79.6% three-month complete response rate and 82.3% DOR at 12 months reinforcing its potential to be the first FDA-approved non-surgical option for treatment of LG-IR-NMIBC,” said Liz Barrett, President and CEO of UroGen. “We estimate 82,000 patients suffer from this highly recurrent disease in the U.S. each year and may benefit from an innovative approach to treating their disease.” The most common treatment-emergent adverse events in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. TEAEs were typically mild-to-moderate in severity. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102.

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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.