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: 

• Axsome Therapeutics (AXSM), 268% surge in interest
• X4 Pharmaceuticals (XFOR), 257% surge in interest
• Heron Therapeutics (HRTX), 227% surge in interest
• Syndax Pharmaceuticals (SNDX), 211% surge in interest
• Avidity Biosciences (RNA), 191% surge in interest
• Cyclacel Pharmaceuticals (CYCC), 157% surge in interest
• Rigel Pharmaceuticals (RIGL), 139% surge in interest
• SpringWorks Therapeutics (SWTX), 136% surge in interest
• Agenus (AGEN), 132% surge in interest
• Seres Therapeutics (MCRB), 128% surge in interest

Pipeline and key clinical candidates for these companies:

Axsome Therapeutics is developing and delivering novel therapies for central nervous system, or CNS, conditions that have limited treatment options. AXS-05 is a novel, oral, patent protected, investigational N-methyl-D-aspartate receptor antagonist with multimodal activity under development for the treatment of Alzheimer’s disease agitation and other central nervous system disorders.

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.

Heron Therapeutics is a commercial-stage biotechnology company that says it is developing “novel, patient-focused solutions that apply its innovative science and technologies to already-approved pharmacological agents for patients suffering from pain or cancer.”

Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Highlights of the company’s pipeline include revumenib, a selective menin inhibitor, and Niktimvo, an FDA-approved monoclonal antibody that blocks the colony stimulating factor 1 receptor.

Avidity Biosciences’ proprietary AOCs – Antibody Oligonucleotide Conjugates – are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics, the company says. Avidity’s advancing and expanding pipeline has three programs in clinical development. AOC 1001 is designed to treat people with myotonic dystrophy type 1 and is currently in Phase 1/2 development with the ongoing MARINA and MARINA-OLE trials. AOC 1020 is designed to treat people living with facioscapulohumeral muscular dystrophy and is currently in Phase 1/2 development with the FORTITUDE trial. AOC 1044 is designed for people with Duchenne muscular dystrophy mutations amenable to exon 44 skipping and is currently in Phase 1/2 development with the EXPLORE44 trial.

Cyclacel is a clinical-stage, biopharmaceutical company developing cancer medicines based on cell cycle, transcriptional regulation, epigenetics and mitosis biology. The transcriptional regulation program is evaluating fadraciclib, a CDK2/9 inhibitor, and the epigenetic/anti-mitotic program plogosertib, a PLK1 inhibitor, in patients with both solid tumors and hematological malignancies. Cyclacel’s strategy is to build a diversified biopharmaceutical business based on a pipeline of novel drug candidates addressing oncology and hematology indications.

Rigel Pharmaceuticals is dedicated to discovering, developing and providing novel small molecule drugs that significantly improve the lives of patients with hematologic disorders, cancer, and rare immune diseases. Rezlidhia is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia, or AML, with a susceptible isocitrate dehydrogenase-1 mutation as detected by an FDA-approved test.

SpringWorks is a commercial-stage biopharmaceutical company applying a precision medicine approach to developing and delivering life-changing medicines for people with severe rare diseases and cancer. Ogsiveo, approved in the United States for the treatment of adult patients with progressing desmoid tumors who require systemic treatment, is SpringWorks’ first FDA-approved therapy.

Agenus is an immuno-oncology company targeting cancer and infectious diseases with a comprehensive pipeline of immunological agents. The company’s mission is to expand patient populations benefiting from cancer immunotherapy through combination approaches, using a broad repertoire of antibody therapeutics, adoptive cell therapies and adjuvants.

Seres Therapeutics is a microbiome therapeutics company developing a novel class of multifunctional bacterial consortia that are designed to functionally interact with host cells and tissues to treat disease. Seres’ SER-109 program achieved “the first-ever positive pivotal clinical results for a targeted microbiome drug candidate,” according to the company, and has obtained Breakthrough Therapy and Orphan Drug designations from the FDA. 

Recent news on these stocks:

November 14

Piper Sandler analyst Allison Bratzel raised the firm’s price target on Rigel Pharmaceuticals to $23 from $15 driven by pipeline developments. The firm keeps a Neutral rating on the shares following quarterly results.

November 13

X4 Pharmaceuticals announced positive new clinical data from its now completed Phase 2 clinical trial evaluating mavorixafor, an oral CXCR4 antagonist, in the treatment of people with chronic neutropenia, or CN. An analysis of final data from the six-month study showed that once-daily oral mavorixafor durably and meaningfully increased participants’ mean absolute neutrophil counts, or ANC. In addition, when tested in combination with injectable granulocyte colony-stimulating factor, or G-CSF, mavorixafor treatment enabled clinicians to substantially reduce G-CSF dosing while maintaining normal mean ANC levels. The Phase 2 study of mavorixafor was a six-month, open-label clinical trial that enrolled a total of 23 participants diagnosed with idiopathic, congenital, or cyclic chronic neutropenia. The analysis presented today includes results from the two study treatment groups: mavorixafor monotherapy and mavorixafor in combination with G-CSF. Consistent with previously presented analyses, results from participants receiving mavorixafor monotherapy showed that mavorixafor durably increased mean ANC from baseline, with mean ANC reaching normal levels at Month 3 and Month 6. Further analysis showed that those with severe CN achieved greater than two-fold increases in mean ANC levels out to six months, reaching levels typically targeted by physicians for patients with severe CN. Mavorixafor in combination with injectable G-CSF: New results presented demonstrated that physicians chose to reduce G-CSF dosing in nine of 12 eligible participants. Of those nine, eight had G-CSF reduced at the earliest timepoint permitted and three were taken completely off of G-CSF prior to their Month 6 visit. Mean reductions in G-CSF were 52% at Month 3 and 70% at Month 6, while mean ANC levels remained in the normal range. The three participants receiving mavorixafor who remained on stable doses of G-CSF maintained mean ANC levels in the normal range at all timepoints. X4 also announced new findings from a sub-study comparing the mean percentage of functional neutrophils in samples from healthy donors to participants from the Phase 2 CN study using two common study methods. These results demonstrated that the mean percentage of functional circulating neutrophils in CN participants in this sub-study was comparable to that of healthy donors after six months of mavorixafor dosing. The subset of participants with congenital neutropenia also had mean percentage of functional neutrophils comparable to those of healthy donors.

Scotiabank analyst George Farmer lowered the firm’s price target on Syndax to $18 from $23 and keeps a Sector Perform rating on the shares. The firm lowered its price target on the stock due to what it views as an increased risk of securing FDA approval of revumenib for the treatment of relapsed-refractory acute myeloid leukemia harboring NPM-1 mutation, the analyst tells investors.

Seres reported lower-than-expected Q3 earnings per share from continuing operations, with the company noting it expects to fund operationgs into 4Q25. Commenting on the results, the company said, “Our SER-155 data provides strong evidence highlighting its potential to significantly reduce the risk of bacterial bloodstream infections, a leading cause of mortality and morbidity in patients undergoing allogeneic hemopoietic stem cell transplants, as well as other medically vulnerable populations. Based on these highly encouraging results, including a relative risk reduction of 77% in BSIs in the active arm as compared to placebo, we have requested Breakthrough Therapy designation and Qualified Infectious Disease Product designation, and anticipate feedback from the FDA by the end of this year. Additionally, we are planning for the next clinical study in allo-HSCT, which we believe could be a single registration study for efficacy. We intend to engage with the agency in the first quarter of 2025 to discuss our clinical study results and future study design.”

November 12

Heron Therapeutics reported Q3 EPS of (3c) against a consensus of (3c), and reported Q3 revenue of $32.81M against a consensus of $36.93M. “We are pleased to report that with our prudent financial management and continued revenue growth we were able to achieve positive adjusted EBITDA for the quarter,” said CEO Craig Collard. “The future looks bright as we continue to grow all product revenue. The CrossLink partnership roll out, FDA approval of the VAN in September, and inclusion in the CMS Final Rule Non-Opioid Policy for Pain Relief, positions ZYNRELEF for significant growth within the surgical setting. We continue to deliver on our commitment to financial efficiency while growing revenue. The team has made great strides in the transformation of Heron over the past year. We are looking forward to a strong Q4 which is off to a great start.” Heron Therapeutics narrowed its FY24 revenue view to $140M-$146M from $138M-$158M, against a consensus of $148.4M.

Syndax announced positive topline results from the relapsed or refractory mutant NPM1 – mNPM1 – acute myeloid leukemia, or AML, cohort in the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib, an oral, small molecule menin inhibitor. The primary endpoint was met with a complete remission, or CR, plus CR with partial hematological recovery rate of 23% among the efficacy evaluable adults with R/R mNPM1 AML in the Phase 2 portion of the AUGMENT-101 trial. Among the patients who achieved CR/CRh, 12 patients had a CR and three had a CRh. The observed median duration of CR/CRh responses was 4.7 months at data cutoff with three patients remaining in response. Overall response rate was 47%. 17% of patients who achieved an overall response underwent hematopoietic stem cell transplant following treatment with revumenib, with three resuming revumenib therapy post-transplant. The safety profile observed with revumenib in this population was consistent with previously reported data. Treatment-related adverse events leading to treatment discontinuations were 5%. Grade 3 treatment-related DS was observed in 11% of patients while 2% experienced Grade 4 DS and no patients experienced Grade 5.

Agenus reported a year-over-year increase in Q3 earnings per share, though revenue for the quarter fell short of Wall Street estimates. “BOT/BAL represents one of the most significant advancements in cancer immunotherapy, showing remarkable results in MSS colorectal cancer where previous treatments have fallen short,” said Garo Armen, Ph.D., Chairman and CEO of Agenus. “Its potential extends beyond this challenging cancer type, with promising efficacy seen in the neoadjuvant setting and other hard-to-treat cancers. While we are excited by these achievements, we remain mindful of the financial challenges that come with advancing such breakthrough therapies.

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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.