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: 

• Oncternal Therapeutics (ONCT), 910% surge in interest
• Scholar Rock (SRRK), 839% surge in interest
• Cabaletta Bio (CABA), 328% surge in interest
• Cassava Sciences (SAVA), 324% surge in interest
• BridgeBio Pharma (BBIO), 252% surge in interest
• Intellia Therapeutics (NTLA), 131% surge in interest

Pipeline and key clinical candidates for these companies:

Oncternal Therapeutics is a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies for the treatment of patients with cancers that have critical unmet medical need. Oncternal pursues drug development targeting promising, yet untapped biological pathways implicated in cancer generation or progression, focusing on hematological malignancies and prostate cancer.

Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Scholar Rock is creating a pipeline of novel product candidates with “the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, and fibrosis,” the company states. Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path, it has noted.

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.

Cassava Sciences is a clinical-stage biotechnology company that says its mission is to detect and treat neurodegenerative diseases, such as Alzheimer’s disease. “Its novel science is based on stabilizing-but not removing-a critical protein in the brain. The company’s product candidates have not been approved by any regulatory authority, and their safety, efficacy or other desirable attributes have not been established,” Cassava has stated.

BridgeBio Pharma is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials, the company has stated.

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.

Recent news on these stocks:

November 25

Shares of Scholar Rock rose recently after rival Biohaven (BHVN) reported that taldefgrobep alpha showed clinically meaningful improvements in motor function at all timepoints on the Motor Function Measurement-32 scale, but the treatment arm did not statistically separate on the primary outcome at Week 48 compared to the placebo+standard of care group, in the RESILIENT SMA study. Previously, Scholar Rock hit on the primary endpoint in the company’s own Phase 3 trial of apitegromab in patients with SMA, or spinal muscular atrophy.

Cassava Sciences announced that the topline results from the Phase 3 ReThink-ALZ study of simufilam in mild-to-moderate AD did not meet each of the pre-specified co-primary, secondary and exploratory biomarker endpoints. The co-primary endpoints were the change in cognition and function from baseline to the end of the double-blind treatment period at week 52, assessed by the ADAS-COG12 and ADCS-ADL scales, comparing simufilam to placebo. Simufilam continued to demonstrate an overall favorable safety profile. The Company will hold a webcast at 8 AM ET. “The results are disappointing for patients and their families who are living with this disease and physicians who have been looking for novel treatment options. We took careful measures to enroll patients with mild-to-moderate AD. Despite that, the loss of cognition in the placebo group was less pronounced than was previously reported in other placebo-controlled studies in AD. We are working to understand this better,” said Rick Barry, President and Chief Executive Officer. “A result like this has implications on our second Phase 3 trial, ReFocus-ALZ. We have made the difficult decision to discontinue ReFocus-ALZ, given the nature of today’s reported results. The complete 52-week dataset will be available from the study along with a large portion of 76-week data. We intend to report detailed analyses of both studies in the future. We will also be discontinuing the Open Label Extension study. We have a special gratitude for the patients and their families and caregivers who participated in our clinical program for AD. We are also immensely grateful to our employees, study investigators and site coordinators, as well as our other partners, for their commitment to this program. We hope the information we have gathered can ultimately be used to benefit ongoing research in AD.” Cassava will continue to review all of the data and evaluate next steps.

Intellia Therapeutics announced that the FDA has granted Regenerative Medicine Advanced Therapy, RMAT, designation to nexiguran ziclumeran for the treatment of hereditary transthyretin amyloidosis with polyneuropathy, ATTRv-PN. Development and commercialization of nex-z is led by Intellia as part of a multi-target collaboration with Regeneron (REGN). “This RMAT designation underscores the transformative potential of nex-z, our investigational in vivo CRISPR-based gene editing therapy for those living with hereditary ATTR amyloidosis with polyneuropathy,” said Intellia President and Chief Executive Officer John Leonard, M.D. “It was granted following the FDA’s review of our compelling interim Phase 1 data that indicated our one-time treatment led to rapid, deep and durable TTR reduction, which is expected to halt and potentially reverse the disease. We look forward to working closely with the FDA to bring this potential paradigm-shifting therapy to patients as quickly as possible.”

November 22

BridgeBio Pharma announced that the FDA approved Attruby, an orally-administered near-complete stabilizer of Transthyretin, or TTR, for the treatment of adults with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization. The FDA approval is based on positive results seen in the ATTRibute-CM Phase 3 study, where Attruby significantly reduced death and cardiovascular-related hospitalization, and improved quality of life. “Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR. Attruby was designed to mimic a naturally occurring ‘rescue mutation’ of the TTR gene that targets the root cause of ATTR-CM, destabilization of the native TTR tetramer. Through near-complete TTR stabilization, Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A and to demonstrate benefit on cardiovascular outcomes,” the company stated. BridgeBio submitted a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025. BridgeBio has granted exclusive rights to Bayer to commercialize acoramidis for ATTR-CM in Europe. “With the landmark approval of Attruby, we gain the ability to serve patients with ATTR-CM. I’m grateful to each trial participant, their families, and the physicians, scientists and our team at BridgeBio who made this possible. Our journey is not over as we look to pursue approvals globally, next in Europe, Japan, and Brazil, and to continue exploring the full potential of this treatment. I am thrilled to extend our mission of ‘putting patients first’ with this third FDA approval in less than 10 years,” said Neil Kumar, Ph.D., BridgeBio founder and CEO.

November 18

Cabaletta Bio announced new and updated clinical data on CABA-201 demonstrating the potential to achieve drug-free, compelling clinical responses based on eight patients dosed across the ongoing Phase 1/2 RESET-Myositis, RESET-SLE and RESET-SSc clinical trials. These data were presented in oral and poster presentations at the American College of Rheumatology Convergence 2024, which is being held at the Walter E. Washington Convention Center in Washington, D.C. from November 14-19, 2024. 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 immunosuppressive therapies in patients with autoimmune diseases. Cabaletta is currently evaluating CABA-201 in the RESET clinical development program across five company-sponsored clinical trials that each have disease-specific cohorts with six patients per cohort. All cohorts are evaluating the same single, weight-based dose of CABA-201 at 1 x 106 cells/kg without a dose escalation requirement. Treatment with CABA-201 in each clinical trial includes a preconditioning regimen of fludarabine and cyclophosphamide, consistent with the dosing regimen used in the third-party academic studies, except for the RESET-PV trial which is evaluating CABA-201 without preconditioning. New and Updated Clinical Data Summary: As of the data cut-off date of November 1, 2024, eight patients had been dosed with CABA-201 with sufficient follow-up to be evaluable across the RESET clinical development program. In the RESET-Myositis trial, one patient in the immune-mediated necrotizing myopathy cohort completed six months of follow-up and two patients, one in the IMNM cohort and one in the dermatomyositis cohort, each completed one month of follow-up. In the RESET-SLE trial, one patient in the non-renal systemic lupus erythematosus cohort completed six months of follow-up, one patient in the lupus nephritis cohort completed four months of follow-up and two patients in the non-renal SLE cohort each completed one month of follow-up. Translational assessments from the third patient in the non-renal SLE cohort were not available for inclusion at the time of the data cut-off. In the RESET-SSc trial, one patient in the severe skin cohort completed six weeks of follow-up. Across these eight patients treated with CABA-201, 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. Safety and Tolerability Profile: CABA-201 has shown a favorable risk-benefit profile in patients with active and refractory autoimmune disease. Through 28 days of follow-up, no evidence of cytokine release syndrome of any grade was observed in five of the eight patients. Low-grade CRS was observed in three patients, all of which recovered following standard care. Tocilizumab was not administered for any cases of CRS. No evidence of immune effector cell-associated neurotoxicity syndrome of any grade has been observed in any patient since reporting the initial safety data on the first LN patient in August 2024. This patient had acute inflammatory events shortly before CABA-201 treatment and demonstrated an abnormal, pro-inflammatory cytokine profile prior to infusion that continued after CABA-201 infusion, suggestive of a possible occult infection. Translational Assessments: CABA-201 induced consistent and complete B cell depletion, with early naive B cell repopulation suggesting the potential to generate an immune system reset CAR T cell expansion associated with CABA-201 reached its peak between day 8 and day 15. Translational assessments from the first patient in the LN cohort indicated a second peak at day 29. Complete B cell depletion was observed by day 22 after CABA-201 infusion. B cell repopulation occurred in the first two patients treated with CABA-201 as early as 8 weeks and exhibited a transitional naive phenotype, reflecting the production of new B cells after deep systemic depletion. Two of the three patients with follow-up beyond three months demonstrated a reduction in disease-associated antibodies. Clinical responses in all three of these patients were observed independent of autoantibody levels. Vaccine and infectious pathogen antibodies remained generally stable. Clinical Outcomes: CABA-201 provided compelling signs of early efficacy, supporting the potential for drug-free clinical responses Initial clinical responses in the RESET-Myositis trial were consistent with published data with response kinetics appearing to differ between myositis subtypes. The first known adult DM patient dosed with CAR T in the form of CABA-201 demonstrated an improvement in muscle strength to normal and a major total improvement score response off all immunosuppressants at one month of follow-up. The Cutaneous Dermatomyositis Disease Area and Severity Index – Activity improved from 25 to 9. At six months of follow-up, the first IMNM patient demonstrated a continued and improved clinical response off immunosuppressants and without flares. At one month of follow-up, the second IMNM patient demonstrated a total improvement score consistent with the first IMNM patient at one month after CABA-201 infusion off immunosuppressants. All four patients in the RESET-SLE trial demonstrated clinical responses off immunosuppressants. All three patients in the non-renal SLE cohort demonstrated no clinical symptoms on SLEDAI-2K as of the latest follow-up and the first patient has completed a prednisone taper to discontinuation. The first patient in the LN cohort, who experienced the previously reported ICANS event, had a SLEDAI that improved from 22 at baseline to 8 at month four of follow-up. The patient’s proteinuria improved more than 90%, approaching normal levels, while off all immunosuppressants and with an ongoing prednisone taper. The first patient in the severe skin cohort in the RESET-SSc trial demonstrated early clinical improvements after discontinuation of disease-specific therapy. The modified Rodnan Skin Score of the first patient in the severe skin cohort improved from 42 at baseline to 36 at day 42, suggesting the potential emergence of a drug-free clinical response.

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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.