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: 

• Biocardia (BCDA), 757% surge in interest
• Mirum Pharmaceuticals (MIRM), 641% surge in interest
• Intellia (NTLA), 585% surge in interest
• NanoString Technologies (NSTG), 273% surge in interest
• Cidara Therapeutics (CDTX), 224% surge in interest
• Ideaya Biosciences (IDYA), 188% surge in interest
• Novavax (NVAX), 184% surge in interest
• Vistagen Therapeutics (VTGN), 178% surge in interest
• Zai Lab (ZLAB), 157% surge in interest
• Ascendis Pharma (ASND), 153% surge in interest

Pipeline and key clinical candidates for these companies:

BioCardia is a developer of cell and cell-derived therapies for cardiovascular and pulmonary disease. The company has two biotherapeutic platforms, CardiAMP autologous bone marrow-derived mononuclear cell therapy for cardiovascular indications and the NK1R+ allogeneic bone marrow-derived mesenchymal stem cell therapies for cardiovascular and pulmonary diseases.

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.

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.

NanoString Technologies says it offers “an ecosystem of innovative discovery and translational research solutions” to map the universe of biology – including the GeoMx Digital Spatial Profiler, the CosMx Spatial Molecular Imager and the AtoMx Spatial Informatics Platform. The CosMx SMI and AtoMx SIP platforms are expected to launch in 2022.

Cidara is developing long-acting therapeutics designed to help improve the standard of care for patients facing serious diseases. The company’s portfolio is comprised of new approaches aimed at transforming existing treatment and prevention paradigms, first with its lead Phase 3 antifungal candidate, rezafungin, in addition to DFCs targeting viral and oncology diseases from Cidara’s proprietary Cloudbreak platform.

Ideaya Biosciences is a synthetic lethality focused precision medicine oncology company committed to the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics. Ideaya says it is applying its research and drug discovery capabilities to synthetic lethality, which “represents an emerging class of precision medicine targets.”

Novavax says it promotes improved health by discovering, developing and commercializing innovative vaccines to protect against serious infectious diseases. Novavax offers a differentiated vaccine platform that combines a recombinant protein approach, innovative nanoparticle technology and Novavax’s patented Matrix-M adjuvant to enhance the immune response.

Vistagen is a late clinical-stage biopharmaceutical company whose clinical-stage candidates are targeting multiple types of anxiety and depression. PH94B and PH10 belong to a new class of drugs known as pherines, which are designed with a novel rapid-onset mechanism of action that activates chemosensory neurons in the nasal passages and can impact the olfactory-amygdala neural circuits without systemic uptake or direct activity on CNS neurons in the brain.

Zai Lab Limited is a commercial-stage biopharmaceutical company based in China and the United States focused on bringing medicines for oncology, autoimmune disorders, infectious diseases, and neurological disorders to patients in China and around the world.

Ascendis Pharma is applying its platform technology to “build a leading, fully integrated, global biopharmaceutical company focused on making a meaningful difference in patients’ lives,” the company states. The company uses its TransCon technologies to “create new and potentially best-in-class therapies,” Ascendis says.

Recent news on these stocks:

September 7

NanoString announced three new Contract Research Organization CosMxTM Spatial Molecular Imager partnerships. Macrogen, Propath UK, and Sirona Dx offer global biopharma customers new access to CosMx SMI technology for translational drug development. These specialty CROs provide biomarker testing services for drug developers and academic research institutions. Korea-based Macrogen, which provides personalized healthcare services to predict and prevent diseases and provide personalized treatment options for patients, is the first CRO in the Asia-Pacific region to offer both CosMx SMI and GeoMx DSP platforms.

September 6

BioCardia announced a clarification and next steps on its autologous CardiAMP cell therapy programs. Based on the recent interim results in the CardiAMP autologous cell therapy for the treatment of heart failure, or BCDA-01, the Company is exploring development of a new Phase III clinical trial protocol. The Finkelstein Schoenfeld, or FS, primary composite endpoint used in the CardiAMP HF Trial has tiers of outcomes in decreasing order of importance: heart death equivalent, major adverse cardiac and cerebrovascular MACCE, and Six Minute Walk Distance, or 6MWD. Since the primary FS endpoint is a composite of these elements, meeting the FS endpoint may be met even if one or more of the components does not individually demonstrate statistical significance. This has implications for the future trial design. The CardiAMP HF Trial interim results showed that the first two most important tier outcomes, occurring in 30% of the study patients, could have contributed sufficiently toward efficacy given a longer follow-up period than one year. If these interim results are replicated in a future study in which the third tier 6MWD is replaced with a more objective endpoint, there may be a pathway to a successful trial for product registration in the U.S. The FDA has previously expressed a preference for replacing the 6MWD with Cardiopulmonary Exercise Testing, which is a more objective outcome.

Cidara Therapeutics has announced that Janssen Pharmaceuticals, one of the Janssen Pharmaceutical Companies of Johnson & Johnson (JNJ), has delivered to Cidara its Election to Proceed Notice for CD388, or JNJ-0953, which is being developed for the universal prevention of influenza A and B. Responsibility for future development, manufacturing and commercialization activities of CD388 will be assumed by Janssen, which intends to transfer its rights and obligations under the agreement to another entity. As part of its exclusive worldwide collaboration with Janssen, Cidara is studying CD388 in Phase 1 and 2a clinical trials evaluating safety and pharmacokinetics as well as efficacy as pre-exposure prophylaxis against an influenza virus in healthy volunteers during a human challenge study. Earlier this year, Cidara announced promising interim efficacy and safety data from the Phase 2a study. The interim analysis demonstrated that a single dose of CD388 decreased viral replication in the upper respiratory tract and lowered influenza incidence rate compared to placebo. Cidara will receive a $7M milestone payment from Janssen for the election to proceed for CD388 and other influenza DFCs. Under the collaboration agreement, Cidara is eligible to potentially receive an additional $685 million in development, regulatory and commercial milestones, plus tiered royalties on worldwide sales.

September 5

Travere Therapeutics (TVTX) and Mirum Pharmaceuticals announced the completion of the previously announced sale of Travere’s bile acid product portfolio that includes Cholbam and Chenodal, two medications addressing rare diseases in high-need settings. In connection with the closing of the Asset Purchase, Travere received an upfront payment of $210 million from Mirum, and remains eligible to receive up to $235 million in potential sales-based milestone payments. Mirum has acquired Travere’s rights to Cholbam, indicated for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease, and Chenodal, indicated for the treatment of radiolucent stones in the gallbladder, which is also under Phase 3 clinical evaluation for cerebrotendinous xanthomatosis. The closing of the Asset Purchase was subject to the satisfaction of customary closing conditions, including the expiration of the waiting period under the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976. In connection with the closing, Travere will provide certain transitional services to Mirum.

Vistagen and Fuji Pharma C announced they have entered into a time-limited agreement to negotiate exclusively with each other regarding a potential license to develop and commercialize Vistagen’s PH80 in Japan, including for the acute treatment of moderate to severe vasomotor symptoms due to menopause and potentially other indications. Vistagen’s PH80 neuroactive nasal spray demonstrated statistically significant efficacy versus placebo in an exploratory double-blind, placebo-controlled Phase 2A study in women diagnosed with menopausal hot flashes. Fuji will make a non-refundable payment of $1.5M to secure the time-limited exclusive negotiation rights for the Japanese market. “As we have seen across our neuroactive pherine nasal spray pipeline, PH80 offers exciting potential to transform a significant segment of a major healthcare market, including the current treatment landscape for women’s healthcare,” said Shawn Singh, CEO of Vistagen. “Menopausal hot flashes affect millions of women worldwide. We share Fuji Pharma’s long-standing commitment to deliver innovative treatment options with potential to enable women to improve their physical, mental and social well-being. As we continue to advance our PH80 development program in the U.S., we look forward to continuing our ongoing discussions with Fuji regarding a potential development and commercialization collaboration in Japan.”

Ascendis Pharma announced new post hoc analysis showing adults with hypoparathyroidism treated with TransCon PTH demonstrated substantial improvement in estimated glomerular filtration rate, eGFR , suggesting improved kidney function. TransCon PTH, palopegteriparatid, is an investigational prodrug of parath yroid hormone PTH 1-34 administered once daily designed to provide sustained release of active PTH within the physiological range for 24 hours per day in adult patients with hypoparathyroidism. “Chronic kidney disease is one of the most consequential complications of chronic hypoparathyroidism and its treatment with conventional therapy. Diminished kidney function is associated with its own set of complications, including hypertension, edema, and weakness,” said Dr. Aliya Khan, M.D., Clinical Professor of Medicine at McMaster University and Director of the Calcium Disorders Clinic at McMaster University Medical Center. “The observations in the post hoc analysis of the PaTHway Trial, suggesting that treatment with TransCon PTH reverses impaired kidney function in patients with hypoparathyroidism, further substantiating the promise for a major improvement in treatment opportunities and outcomes for people living with hypoparathyroidism.””We will continue to study this important data and topic, which suggests the potential of TransCon PTH to address physician and patient concerns about soft-tissue calcifications and decreased kidney function associated with conventional therapy,” said Aimee Shu, M.D., Vice President, Clinical Development, Endocrine & Rare Diseases at Ascendis Pharma. “In addition to reducing risk for patients, this could also help significantly reduce the healthcare burden associated with hypoparathyroidism. We look forward to presenting detailed results at an upcoming medical conference.””We will continue to study this important data and topic, which suggests the potential of TransCon PTH to address physician and patient concerns about soft-tissue calcifications and decreased kidney function associated with conventional therapy,” said Aimee Shu, M.D., Vice President, Clinical Development, Endocrine & Rare Diseases at Ascendis Pharma. “In addition to reducing risk for patients, this could also help significantly reduce the healthcare burden associated with hypoparathyroidism. We look forward to presenting detailed results at an upcoming medical conference.”

August 30

Zai Lab announced that the Center for Drug Evaluation, CDE, of the National Medical Products Administration, NMPA, granted Breakthrough Therapy Designation, BTD, for investigational repotrectinib for the treatment of patients with advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with TRK tyrosine kinase inhibitors. The Breakthrough Therapy Designation for repotrectinib was supported by data from both global and Chinese NTRK-positive TKI-pretreated patients enrolled in the Phase 1/2 TRIDENT-1 study. “We are excited to receive our fourth Breakthrough Therapy Designation for repotrectinib in China. Today’s recognition further supports repotrectinib as a potential first-in-class treatment for patients with NTRK-positive, TKI-pretreated solid tumors in China,” said Rafael G. Amado, M.D., President, Head of Global Oncology Research and Development at Zai Lab. “NTRK is estimated to be an oncogenic driver in approximately 0.5 percent of patients with a variety of advanced solid tumors.1 There remains an unmet medical need for NTRK-positive, TKI-pretreated advanced solid tumor patients where there are no targeted therapies currently approved. We look forward to working with regulatory authorities in China to bring this important medicine to patients in need as soon as possible.”

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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.