Stay ahead of biotech stocks seeing a surge in interest from retail and financial professional investors with this exclusive recap from The Fly These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include:
Pipeline and key clinical candidates for these companies:
Avidity Bioscience's 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.
Telesis Bio says it is "empowering scientists with the ability to create novel, synthetic biology-enabled solutions for many of humanity's greatest challenges." The Gibson Assembly method and the first commercial automated benchtop DNA and mRNA synthesis system enable rapid, accurate and reproducible writing of DNA and mRNA for numerous downstream markets, the company has stated.
Mereo BioPharma is a biopharmaceutical company focused on the development of innovative therapeutics for rare diseases. The company has two rare disease product candidates, setrusumab for the treatment of Osteogenesis Imperfecta, or OI, and alvelestat for the treatment of severe alpha-1-antitrypsin deficiency-associated lung disease and Bronchiolitis Obliterans Syndrome.
Sage Therapeutics is a biopharmaceutical company "fearlessly leading the way to create a world with better brain health." The company's mission is to "pioneer solutions to deliver life-changing brain health medicines, so every person can thrive," Sage has stated.
Tracon is a clinical-stage biopharmaceutical company that says utilizing "a cost-efficient, CRO-independent, product development platform" it is advancing its pipeline of novel targeted cancer therapeutics and partnering with other life science companies. The company's clinical-stage pipeline includes Envafolimab, a PD-L1 single-domain antibody given by rapid subcutaneous injection that is being studied in the pivotal ENVASARC trial for sarcoma; YH001, a potential best-in-class CTLA-4 antibody in Phase 1 development; and TRC102, a Phase 2 small molecule drug candidate for the treatment of lung cancer.
Jazz Pharmaceuticals is a biopharmaceutical company who says its purpose is to "innovate to transform the lives of patients and their families." The company has a diverse portfolio of marketed medicines and novel product candidates, from early- to late-stage development, in neuroscience and oncology. Within these therapeutic areas, Jazz says it is "identifying new options for patients by actively exploring small molecules and biologics, and through innovative delivery technologies and cannabinoid science."
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.
Dynavax is a commercial-stage biopharmaceutical company that says it is "developing and commercializing innovative vaccines to help protect the world against infectious diseases." The company has two commercial products, HEPLISAV-B vaccine, which is approved in the U.S., the European Union and Great Britain for the prevention of infection caused by all known subtypes of hepatitis B virus in adults 18 years of age and older, and CpG 1018 adjuvant, currently used in multiple adjuvanted COVID-19 vaccines.
Madrigal is a biopharmaceutical company pursuing novel therapeutics for non-alcoholic steatohepatitis, also known as metabolic dysfunction associated steatohepatitis. Madrigal has advanced its once daily, oral, liver-directed thyroid hormone receptor β-selective agonist, into multiple Phase 3 clinical trials in NASH. Based on Phase 3 results reported to date, the U.S. FDA granted accelerated approval for the therapy for the treatment of adults with NASH with moderate to advanced liver fibrosis.
Recent news on these stocks:
June 12
Avidity Biosciences announced positive initial AOC 1020 data from the Phase 1/2 FORTITUDE trial demonstrating unprecedented and consistent reductions of greater than 50% in DUX4 regulated genes, trends of functional improvement, and favorable safety and tolerability in people living with facioscapulohumeral muscular dystrophy, or FSHD. Avidity plans to accelerate initiation of registrational cohorts in the FORTITUDE study. Avidity also announced delpacibart braxlosiran as the approved international nonproprietary name of AOC 1020, abbreviated as del-brax. Del-brax is the first investigational therapy designed to treat the underlying cause of FSHD, which is caused by the abnormal expression of a gene called double homeobox 4 or DUX4. FSHD is a rare, hereditary disorder marked by life-long, relentless loss of muscle function, significant pain, fatigue, and progressive disability. Currently, there are no approved therapies for the treatment of FSHD. "As the first therapy to directly target DUX4, it is very encouraging to see that the del-brax data demonstrate consistent reductions in DUX4 regulated genes and provided trends of functional improvement in patients with FSHD at the four-month timepoint. These early data would support the notion that del-brax has the potential to change the course of disease for people living with FSHD," said Jeffrey M. Statland, Professor of Neurology, University of Kansas Medical Center, and FORTITUDE trial investigator. "Early data showing trends in del-brax to improve muscle strength and function are very encouraging for patients with FSHD who are in need of treatments to prevent the muscle weakness and disability that is associated with this relentlessly, progressive disease." The AOC 1020 initial data will be highlighted in an oral presentation at the 31st Annual FSHD Society International Research Congress, being held June 13-14, 2024, in Denver, Colorado.
June 11
Ultragenyx Pharmaceutical (RARE) and Mereo BioPharma Group announced positive 14-month results from the Phase 2 portion of the ongoing Phase 2/3 Orbit study demonstrating that, as of a May 24 data cut-off date, treatment with setrusumab continued to significantly reduce incidence of fractures in patients with OI with at least 14 months of follow-up. Treatment with setrusumab also resulted in ongoing and meaningful improvements in lumbar spine bone mineral density, or BMD, at month 12 without evidence of plateau. The large reduction in annualized radiologically confirmed fracture rate previously reported in patients treated for a minimum of 6 months was sustained in patients treated for at least 14 months with a high degree of significance. The median annualized rate of radiologically confirmed fractures across all 24 patients in the 2 years prior to treatment was 0.72. Following a mean treatment duration period of 16 months, the median annualized fracture rate was reduced 67% to 0.00. The annualized fracture rate excluded morphometric vertebral fractures and fractures of the fingers, toes, skull, and face, consistent with the Phase 3 study primary efficacy endpoint. The reduction in annualized fracture rates was associated with continued, clinically meaningful increases in BMD. Tests conducted at the 12-month timepoint demonstrated that treatment with setrusumab resulted in a mean increase in lumbar spine BMD from baseline of 22% across all age groups (5 to less than 26 years old), a further improvement from 14% observed at 6 months of treatment. This increase in BMD is reflected in the change from the mean baseline lumbar spine BMD Z-score of -1.73 to -0.49 at 12 months across all age groups, a substantial normalization in Z-score of +1.25. This is further improved from the mean 6-month Z-score change of +0.85. The improvements in BMD and Z-scores were significant and consistent across all OI sub-types studied.
Sage Therapeutics announced topline results from the Phase 2 SURVEYOR Study. The study met its primary endpoint demonstrating a statistically significant difference between healthy participants and participants with Huntington's Disease - HD - prior to any treatment with dalzanemdor or placebo. SURVEYOR was a small study with three objectives: to determine the magnitude of cognitive impairment in HD compared to healthy participants; to evaluate the safety of dalzanemdor in participants with HD; and to better understand the relationship between changes in cognition and changes in function. Dalzanemdor was generally well-tolerated and no new safety signals were observed. A total of 11 participants with HD experienced treatment emergent adverse events, the vast majority of which were mild to moderate in severity. There were no discontinuations related to TEAEs. There was a small numerical difference observed between dalzanemdor and placebo on the HD-CAB composite score at Day 28. Other prespecified analyses suggested the potential for directionally positive signals in a number of individual component tests of the HD-CAB and in some functional assessments. Additional work is ongoing to further analyze and understand the data including the relationship of changes in cognition to changes in function. The company expects dalzanemdor program milestones in late 2024: report topline data from LIGHTWAVE Study in mild cognitive impairment and mild dementia in Alzheimer's Disease, and report topline data from DIMENSION Study in cognitive impairment associated with HD.
Tracon Pharmaceuticals announced the publication of Phase 2 clinical data of its DNA damage repair inhibitor drug candidate, TRC102, in patients with glioblastoma in Clinical Cancer Research. The article, entitled, "Evaluating the Base Excision Repair Inhibitor TRC102 and Temozolomide for patients with Recurrent Glioblastoma in the Phase 2 Adult Brain Tumor Consortium Trial BERT," highlights the activity of TRC102 given in combination with Temodar chemotherapy in patients with recurrent glioblastoma. TRC102 was evaluated in a Phase 2 trial in combination with Temodar in 19 patients with progressive or recurrent glioblastoma following surgical resection, Temodar and external beam radiotherapy. Extended survival was observed in two patients, both of whom demonstrated significantly enriched signatures of DNA damage response, chromosomal instability, and cellular proliferation by RNA sequencing prior to initiating treatment with Temodar and TRC102. The study was completed by the Adult Brain Tumor Consortium and led by Manmeet Alhuwalia, MD while he was Chair of Neuro-Oncology at Cleveland Clinic prior to his appointment as Chief of Medical Oncology, Chief Scientific Officer, and Deputy Director of the Miami Cancer Institute. The authors concluded the study findings confirm the safety and feasibility of TRC102 given with Temodar for recurrent glioblastoma patients and warrant further evaluation of combination therapy in biomarker-enriched trials enrolling glioblastoma patients with baseline hyperactivated DDR pathways. "We believe that the data generated to date provides a strong rationale for studying TRC102 in combination with Temodar and radiotherapy in newly diagnosed patients with malignant glioma," said James Freddo, M.D., Chief Medical Officer of TRACON. "The Clinical Cancer Research publication supports prior data published in Cancer Cell that patients whose cancers demonstrate activation of DDR pathways may be particularly sensitive to the pharmacologic effects of TRC102." Based on a 100% response rate in a Phase 1 clinical trial combining TRC102 with pemetrexed, cisplatin and radiation therapy in 15 patients with stage III non-squamous non-small cell lung cancer, TRC102 is currently being studied in a randomized Phase 2 clinical trial in combination with chemotherapy and radiation therapy for stage III non-squamous non-small cell lung cancer. This trial is sponsored by the National Cancer Institute through a Cooperative Research and Development Agreement. Determination of the primary endpoint of progression free survival is expected in 2025. TRACON and the NCI have a longstanding history of partnership to develop TRC102, whereby the NCI has funded six Phase 1 or Phase 2 trials through the CRADA.
In a recently published report, Culper Research said it is short Axsome Therapeutics and sees shares "headed lower" as it believes the company's "launch of Auvelity for major depressive disorder has been aided by undisclosed consignment deals with dodgy mail-order pharmacies that subvert prior authorizations, inflating script counts and reported revenues." "In short, Axsome has simply not been paid for 43% of its reported Auvelity revenues since launch. Tellingly, Axsome dismissed its longtime auditor E&Y in June 2023, and in both of its past two annual reports, the Company disclosed critical audit matters related to revenue recognition," the report reads. "We believe that to avoid this burden of PAs at standard pharmacies, Axsome has engaged in an undisclosed program that both consigns Auvelity to a network of dodgy mail-order pharmacies then steers prescriptions towards those pharmacies. These pharmacies then use Axsome's copay cards to fill Auvelity prescriptions prior to even submitting the claim to payors, let alone obtaining an approved PA. This raises anti-kickback concerns, false claims concerns, and revenue recognition concerns, in our view."
June 10
Wolfe Research initiated coverage of Madrigal Pharmaceuticals with an Outperform rating and $382 price target. The company has "only one critical driver" — how well its Rezdiffra performs on launch, the analyst told investors in a research note. The firm is bullish on the drug as it is the first oral drug with no competition and offers "excellent pharmacy strategy", which unintendedly creates economic incentives for PBMs to cover Rezdiffra broadly. Akero (AKRO) is the strongest upcoming competitor, but Madrigal is about four years ahead of it with its Rezdiffra launch and can create a strong moat, Wolfe added.
June 5
Goldman Sachs initiated coverage of Jazz Pharmaceuticals with a Buy rating and $169 price target. Jazz has a diversified global portfolio of commercial assets across two verticals of neuroscience and oncology, with the company on track to achieve its "Vision 2025" goal of $5B revenue, the analyst told investors in a research note. The firm says that while the investor debate has been on the outlook for the company's sleep business, it is optimistic on Jazz's emerging pipeline to support its longer-term growth profile. It sees the stock as attractive at current levels.
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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.
