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: 

• Intra-Cellular Therapies (ITCI), 738% surge in interest
• Mustang Bio (MBIO), 114% surge in interest
• Mirum Pharmaceuticals (MIRM), 86% surge in interest
• Sarepta Therapeutics (SRPT), 60% surge in interest
• Ardelyx (ARDX), 52% surge in interest
• Ovid Therapeutics (OVID), 32% surge in interest

Pipeline and key clinical candidates for these companies:

Intra-Cellular Therapies is a biopharmaceutical company working to understand how therapies affect the inner-workings of cells in the body. The company leverages its intracellular approach to “develop innovative treatments for people living with complex psychiatric and neurologic diseases,” it states.

Mustang Bio is a clinical-stage biopharmaceutical company that says it is “focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers.” Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with medical institutions to advance the development of CAR-T therapies, the company notes.

Mirum Pharmaceuticals is focused on the treatment of rare liver diseases. Mirum’s approved medication is Livmarli , or maralixibat oral solution, which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older. In Europe, the European Committee for Medicinal Products for Human Use has issued a positive opinion for Livmarli for the treatment of cholestatic pruritus in patients with Alagille syndrome two months of age and older. A decision by the European Commission is expected by year-end 2022. Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults.

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.

Ardelyx says it was founded with “a mission to discover, develop and commercialize innovative, first-in-class medicines that meet significant unmet medical needs.” Ardelyx’s first approved product, Ibsrela is available in the United States and Canada. Ardelyx is developing Xphozah, a novel product candidate to control serum phosphorus in adult patients with chronic kidney disease on dialysis, which has completed three successful Phase 3 trials. Ardelyx has a Phase 2 potassium lowering compound, RDX013, for the potential treatment of elevated serum potassium, or hyperkalemia, a problem among certain patients with kidney and/or heart disease and an early-stage program in metabolic acidosis, a serious electrolyte disorder in patients with CKD.

Ovid Therapeutics is striving to conquer seizures and intractable brain disorders with science. The company is advancing a focused pipeline of targeted small molecule candidates to modulate the intrinsic and extrinsic factors involved in neuronal hyperexcitation, which can cause seizures and other neuropathological symptoms. Ovid is developing: OV888, a potent and highly selective ROCK2 inhibitor, for the potential treatment of lesions associated with cerebral cavernous malformations; OV329, a GABA-aminotransferase inhibitor, for the potential treatment of treatment-resistant seizures; and OV350, a direct activator of the KCC2 transporter, for the potential treatment of epilepsies. In addition, the company’s ROCK2 inhibitor and KCC2 activator portfolios have the potential to treat other neurological conditions. Ovid also maintains a significant financial interest in the future regulatory development and potential commercialization of soticlestat, which Takeda is responsible for advancing globally. Soticlestat is a cholesterol 24-hydroxylase inhibitor, which is currently in Phase 3 trials for Dravet and Lennox-Gastaut syndromes.

Recent news on these stocks:

June 20

Sarepta Therapeutics announced FDA approval of an expansion to the labeled indication for Elevidys to include individuals with Duchenne muscular dystrophy, DMD, with a confirmed mutation in the DMD gene who are at least 4 years of age. Confirming the functional benefits, the FDA granted traditional approval for ambulatory patients. The FDA granted accelerated approval for non-ambulatory patients. Continued approval for non-ambulatory Duchenne patients may be contingent upon verification of clinical benefit in a confirmatory trial. Elevidys is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. “Representing many years of dedicated research, development, investment and creative energy, the expansion of the Elevidys label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a defining moment for the Duchenne community. Today also stands as a watershed occasion for the promise of gene therapy and a win for science,” said Doug Ingram, CEO of Sarepta.

June 18

Intra-Cellular Therapies announced positive topline results from Study 502 evaluating lumateperone 42 mg as an adjunctive therapy to antidepressants for the treatment of MDD. This trial, in conjunction with our previously reported positive Phase 3 study, Study 501, forms the basis for our lumateperone sNDA for the adjunctive treatment of MDD. We expect to submit this sNDA to the U.S. Food and Drug Administration FDA n the second half of 2024.”We are confident that the efficacy results from Studies 501 and 502, along with the favorable safety and tolerability profiles from these studies, will make lumateperone a drug of choice for patients suffering with MDD who are having an inadequate response to antidepressant therapy,” said Dr. Sharon Mates, Chairman and CEO of Intra-Cellular Therapies. …These results, further support our vision for CAPLYTA to become a leading option for patients and providers across mood disorders.”…Supplemental NDA sNDA submission for the adjunctive treatment of major depressive disorder MDD anticipated in the second half of 2024…

Oppenheimer downgraded Ovid Therapeutics to Perform from Outperform. The firm noted that Ovid recently reported that partner Takeda (TAK) failed to hit statistical significance on the primary endpoint in both its pivotal Phase 3 studies of Soticlestat in Dravet syndrome and Lennox-Gastaut syndrome. In DS, the study just missed its primary endpoint but showed clinically meaningful and significant effects in multiple secondary EPs. In LGS, the primary endpoint wasn’t hit. Despite Takeda’s decision to move forward and discuss the totality of the data with regulatory bodies, Oppenheimer has opted to take a conservative route for the time being and move to the sidelines given the lack of clarity.

June 17

Mustang Bio announced that updated data from the ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T-cell therapy, show a favorable safety and efficacy profile in patients with Waldenstrom macroglobulinemia a rare form of blood cancer. MB-106 is being developed in a collaboration between Mustang and Fred Hutch Cancer Center to treat patients with relapsed or refractory B-cell non-Hodgkin lymphomas and chronic lymphocytic leukemia. The updated results from the single-institution Phase 1/2 clinical trial were presented during a poster session at the European Hematology Association 2024 Hybrid Congress by Brian Till, M.D., Associate Professor and physician at Fred Hutch and University of Washington. All ten patients in the study were previously treated with Bruton’s tyrosine kinase inhibitors, and their disease continued to progress while on BTKi. Overall, 90% of the patients treated with MB-106 responded to treatment, including 3 complete responses, 2 very good partial responses and 4 partial responses. In addition, 1 patient experienced stable disease. One of the patients who achieved a complete response has remained in remission for 31 months, with an immunoglobulin M level that decreased rapidly to the normal range after treatment with MB-106 and has remained normal since. Patients had a median of nine prior lines of therapy and only one patient has started additional anti-WM treatment after being treated with MB-106. From a safety perspective, cytokine release syndrome occurred in nine patients: five patients with grade 1 and four patients with grade 2. One patient experienced grade 1 immune effector cell-associated neurotoxicity syndrome. No grade 3 or 4 CRS or grade 2, 3 or 4 ICANS has been observed, despite dose escalation.

Mirum Pharmaceuticals announced interim results from two Phase 2b studies evaluating volixibat, an oral ileal bile acid transporter inhibitor in patients with primary biliary cholangitis, or PBC, and primary sclerosing cholangitis, or PSC. Interim results from the VANTAGE study evaluating volixibat in patients with PBC demonstrated a statistically significant improvement in pruritus for volixibat and a placebo-adjusted difference of -2.32 points in the primary endpoint, as measured by the Adult ItchRO scale. 75% of patients on volixibat achieved a greater than 50% reduction in serum bile acids. In addition, there was a significant improvement in fatigue at week 16 with volixibat compared to placebo. No new safety signals were observed, and adverse events were similar between the 20 mg and 80 mg treatment groups. The most common adverse event was diarrhea with all cases mild to moderate, and mostly transient; one case resulted in discontinuation. Four patients experienced serious adverse events, including one in the placebo arm. There were no clinically meaningful changes in liver biomarkers. Based on these results, the VANTAGE PBC trial will continue with a volixibat dose of 20 mg twice daily. Concurrently, the interim analysis for the VISTAS PSC study was conducted and the independent data review committee recommended that the study continue with the selected volixibat dose of 20 mg twice daily, with no changes to the study. The criteria for continuation included safety as well as a predefined threshold for efficacy. The sponsor and investigators are blinded to the interim results and analysis. “The interim data from the VANTAGE study provide outstanding results in relation to what has been shown for treatment of pruritus in PBC,” said Joanne Quan, MD, chief medical officer at Mirum. “With both VISTAS and VANTAGE advancing to enroll their confirmatory portions, we are excited about volixibat as a potential future option to help patients overcome one of the most prevalent and burdensome symptoms of these rare liver diseases.”

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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.