Biotech Alert: Searches spiking for these stocks today

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: 

• Krystal Biotech (KRYS), 443% surge in interest
• Blueprint Medicines (BPMC), 338% surge in interest
• Avidity Biosciences (RNA), 327% surge in interest
• Achieve Life Sciences (ACHV), 322% surge in interest
• Outlook Therapeutics (OTLK), 290% surge in interest

Pipeline and key clinical candidates for these companies:

Krystal Biotech is a biotechnology company focused on developing and commercializing genetic medicines for patients with rare diseases. The company’s pipeline is based on its proprietary redosable HSV vector. In May, Vyjuvek was approved by the FDA for the treatment of dystrophic epidermolysis bullosa. Vyjuvek is a non-invasive, topical, redosable gene therapy designed to treat DEB at the molecular level.

Blueprint Medicines is a global precision therapy company that says it "creates medicines that selectively target genetic drivers, with the goal of staying one step ahead across stages of disease." Blueprint is delivering its approved medicines to patients in the United States and Europe, and is globally advancing multiple programs for systemic mastocytosis, lung cancer, breast cancer and other genomically defined cancers, and cancer immunotherapy, the company states.

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.

Achieve’s focus is to address the global smoking health and nicotine addiction epidemic through the development and commercialization of cytisinicline, a plant-based alkaloid with a high binding affinity to the nicotinic acetylcholine receptor. Cytisinicline is an investigational product candidate being developed for treatment of nicotine addiction and has not been approved by the FDA for any indication in the United States.

Outlook Therapeutics is a biopharmaceutical company working to develop and launch ONS-5010/ LYTENAVA as the first FDA-approved ophthalmic formulation of bevacizumab for use in retinal indications, including wet AMD, DME and BRVO. The FDA accepted Outlook Therapeutics’ BLA submission for ONS-5010 to treat wet AMD with a PDUFA goal date of August 29, 2023. The submission is supported by Outlook Therapeutics’ wet AMD clinical program, which consists of three clinical trials: NORSE ONE, NORSE TWO, and NORSE THREE. If ONS-5010 ophthalmic bevacizumab is approved, Outlook Therapeutics expects to commercialize it as the first and only FDA-approved ophthalmic formulation of bevacizumab for use in treating retinal diseases in the United States, United Kingdom, Europe, Japan, and other markets.

Recent news on these stocks:

May 25

Achieve Life Sciences entered into a securities purchase agreement with certain institutional investors providing for the purchase and sale of 3M shares of common stock at a price of $5.50 per share in a registered direct offering. The aggregate gross proceeds from this offering are expected to be approximately $16.5M, prior to deducting placement agent fees and estimated offering expenses. The company intends to use the proceeds from the offering to fund product development and regulatory activities, and for working capital and general corporate purposes. The offering is expected to close on or about May 30, 2023, subject to customary closing conditions. Current Achieve investors, including investors in Dialectic Life Sciences and various entities affiliated with the Hunt Family, as well as members of Achieve’s management, are participating in this transaction. Lake Street Capital Markets is acting as the exclusive placement agent in connection with this offering.

May 23

Blueprint Medicines announced that NEJM Evidence, a journal from The New England Journal of Medicine Group, has published detailed results from the PIONEER trial of AYVAKIT in patients with indolent systemic mastocytosis. Data reported in the publication show that AYVAKIT achieved statistically significant and clinically meaningful benefits across efficacy measures including disease symptoms and pathological mast cell burden, which continued to improve over time through 48 weeks. In addition, AYVAKIT demonstrated a favorable safety profile relative to placebo. On May 22, 2023, AYVAKIT became the first and only medicine approved by the U.S. Food and Drug Administration for the treatment of adults with ISM. AYVAKIT was designed to potently and selectively inhibit KIT D816V, the primary underlying driver of the disease. ISM represents the vast majority of cases of systemic mastocytosis, a rare hematologic disorder that can lead to debilitating symptoms across multiple organ symptoms and a profound impact on patients’ quality of life. PIONEER is a randomized, double-blind, placebo-controlled trial in which 141 patients received AYVAKIT 25 mg once daily plus best supportive care and 71 patients received placebo plus best supportive care. Disease symptoms were assessed using the ISM Symptom Assessment Form. As previously reported, AYVAKIT met the primary and all key secondary endpoints, which were evaluated from baseline to 24 weeks, and showed deepening clinical efficacy through 48 weeks of treatment. Overall, 96 percent of patients with AYVAKIT completed 24 weeks of treatment and opted to cross over to the open-label extension study. Key published results include: Patients treated with AYVAKIT achieved rapid, durable and statistically significant reductions on all measures of pathological mast cell burden, including serum tryptase, KIT D816V variant allele fraction and bone marrow mast cells. AYVAKIT achieved statistically significant and clinically meaningful benefits in overall symptoms – as measured by the ISM-SAF Total Symptom Score – compared to placebo at 24 weeks, with improvements deepening through 48 weeks. In addition, AYVAKIT showed improvements over placebo on the most severe symptom and across all individual symptoms measured. AYVAKIT showed clinically meaningful improvements in multiple exploratory endpoints of quality of life, including a disease-specific measure – Mastocytosis Quality of Life Questionnaire- and general health status measures such as European Quality of Life Visual Analogue Scale, Patient Global Impression of Severity and 12-Item Short-Form Health Survey. AYVAKIT showed a favorable safety profile, with most adverse events mild to moderate in severity. The serious AE rate was 5.0 percent for AYVAKIT and 11.3 percent for placebo. The most common treatment-emergent AEs for AYVAKIT were flushing, edema, increased blood alkaline phosphatase and insomnia.

May 22

Krystal Biotech said it sees cash runway through the end of 2026. "We are extremely pleased to have the support of this strong investor group," said Krish S Krishnan, CEO of Krystal Biotech. "These additional funds, together with our existing cash, cash equivalents and investments, should allow us to fund the VYJUVEK launch, future operations, and the advancement of our growing pipeline through the end of 2026. It also allows us to retain the optionality of monetizing the Priority Review Voucher we received in connection with the FDA approval of VYJUVEK at a future date with favorable terms and without any dilution."

Evercore ISI upgraded Avidity Biosciences to Outperform from In Line with an unchanged price target of $20. The shares have declined to a valuation that the firm believes "adequately reflects" the safety risk for lead program AOC1001 for DM1, while the company recently indicated that the partial hold on AOC1001 is being partially lifted, says the analyst, who sees a path toward "generating a meaningful amount of safety data in a short period of time to better characterize the risk of ischemic events, if any," with AOC1001.

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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.

Keywords: biotech, biotech sector, biotech alert, investingchannel, XBI, SPDR S&P Biotech ETF