The Daily Biotech Pulse: Sarepta Gene Therapy Data, Idera Plunges On Failed Skin Cancer Study, 3 IPOs

Here's a roundup of top developments in the biotech space over the last 24 hours.

Scaling The Peaks

(Biotech Stocks Hitting 52-week Highs March 18)

• AngioDynamics, Inc. ANGO
• Anixa Biosciences, Inc. ANIX
• ENDRA Life Sciences Inc. NDRA (announced a collaboration with Hepion Pharmaceuticals, Inc. HEPA for NASH studies)
• Harvard Bioscience, Inc. HBIO
• Neos Therapeutics, Inc. NEOS (reacted to the shareholder meeting held to vote on the proposal to merge with Aytu BioScience, Inc. AYTU)
• NuVasive, Inc. NUVA
• SI-BONE, Inc. SIBN
• Sierra Oncology, Inc. SRRA
• Silverback Therapeutics, Inc. SBTX
• Terns Pharmaceuticals, Inc. TERN
• Varian Medical Systems, Inc. VAR
• Vericel Corporation VCEL
• Vincerx Pharma, Inc. VINC

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows March 18)

• Foghorn Therapeutics Inc. FHTX (issued a corporate update)
• Galecto, Inc. GLTO
• Voyager Therapeutics, Inc. VYGR

Stocks In Focus

Sarepta Presents Positive Data For Gene Therapy to Treat Limb-gridle Muscular Dystrophy

Sarepta Therapeutics, Inc. SRPT announced new results from the ongoing study of SRP-9003, its investigational gene therapy for limb-girdle muscular dystrophy Type 2E, showing sustained protein expression in muscle tissue.

Functional assessments taken two years following treatment in low-dose arm and one year after treatment in high-dose arm showed that patients continued to demonstrate stability in their NSAD total score and improvements on timed function tests.

Results in both cohorts continue to reinforce the safety and tolerability profile of SRP-9003, the company said.

The results were presented today at the 2021 Muscular Dystrophy Association Annual Clinical and Scientific Conference.

In after-hours trading, the stock added 4.75% to $87.75.

Cyclerion Announces Departure of Chief Medical Officer

Cyclerion Therapeutics, Inc. CYCN said Christopher Wright, its chief medical officer, has resigned his position with the company. Wright will provide Cyclerion with transitional support until March 26, and will also join the company's Clinical Advisory Board.

vTv Commences Study to Evaluate Potential of TTP399 In Reducing Ketoacidosis In Type 1 Diabetes Patients

vTv Therapeutics Inc. VTVT announced the initiation of a mechanistic study exploring the effects of TTP399 on ketone body formation during a period of insulin withdrawal in people with type 1 diabetes. The phase 1 study, the company said, will test the hypothesis that treatment with vTv's TTP399 will not result in the increased production of ketones, a precursor to ketoacidosis, which can lead to hospitalization in patients with type 1 diabetes.

Diabetic ketoacidosis is a serious complication of diabetes that occurs when the body produces high levels of blood acids called ketones when insulin production by the body is inadequate.

The stock lost 3.91% to $2.70 in after-hours trading.

Incyte's Jakafi Flunks a Late-stage Study In Treating ARDS In COVID-19 Patients

Incyte Corporation INCY said the Phase 3 DEVENT study evaluating the efficacy and safety of Jakafi, 5mg and 16mg doses, plus standard-of-care versus SoC in patients on mechanical ventilation with COVID-19 associated Acute Respiratory Distress Syndrome did not meet its primary endpoint.

Improvement in mortality for each dose compared to placebo, while trending toward positive, was not statistically significant for the overall study population, the company said. However, significant improvement in mortality was seen in U.S. study participants at both doses, and in the overall population when data from both treatment arms was pooled.

The company said it will discuss with the FDA regarding an expanded access program to allow eligible patients in the U.S. with COVID-19-associated ARDS to receive ruxolitinib.

In after-hours trading, the stock rose 1.06% to $79.08 in after-hours trading.

Idera Says Lead Drugs Flunks Late-Stage Skin Cancer Study

Idera Pharmaceuticals, Inc. IDRA said the ILLUMINATE-301 pivotal registration trial of its lead product candidate tilsotolimod in combination with Bristol-Myers Squibb Company's BMY Yervoy versus ipilimumab alone in patients with anti-PD-1 refractory advanced melanoma, did not meet its primary endpoint of objective response rate.

Idera said it is evaluating its next steps regarding continuation of the trial toward its overall survival endpoint, which includes evaluating the full data set when it is available. The company also plans to continue its ILLUMINATE-206 Phase 2 study of tilsotolimod in combination with ipilimumab and nivolumab in patients with microsatellite stable colorectal cancer.

The stock plunged 59.88% to $2.01 in after-hours trading.

Merck, Eisai Report Positive Late-Stage Data For Combo Therapy In Endometrial Cancer

Merck MRK and Eisai Co., Ltd. ESALY announced positive data from the pivotal Phase 3 KEYNOTE-775/Study 309 trial that evaluated the combination of the former's Keytruda plus the latter's Lenvima, for the treatment of certain patients with advanced, metastatic or recurrent endometrial cancer following one prior platinum-based regimen in any setting.

The data was presented in an oral plenary session at the virtual Society of Gynecologic Oncology 2021 Annual Meeting on Women's Cancer.

Kiniksa Announces FDA Approval for Arcalyst In Treating Autoinflammatory Cardiovascular Disease

Kiniksa Pharmaceuticals, Ltd. KNSA said the FDA approved Arcalyst, a weekly, subcutaneously-injected, recombinant fusion protein, for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and children 12 years and older. The company expects to launch the drug in April 2021.

The stock gained 10.50% to $24 in after-hours trading.

Marker Therapeutics Gains On Insider Buying

Marker Therapeutics, Inc. MRKR shares moved to the upside after a series of filings by the company revealed company insiders bought shares in the company.

The stock was up 21.11% to $2.18 in after-hours trading.

Earnings

Alpine Immune Sciences, Inc. ALPN reported an increase in its fourth-quarter revenues from $884,000 in 2019 to $5.64 million in 2020. The net loss per share narrowed from 33 cents to 27 cents.

The stock slipped 5.87% to $13 in after-hours trading.

On The Radar

Clinical Readouts

The Society of Gynecologic Oncology Meeting Presentations

Aravive, Inc. ARAV: Updated data from the Phase 1b trial evaluating AVB-500 in platinum resistant ovarian cancer (3:35 pm); Additionally, a trial in progress poster is to be presented on the Phase 1/2 investigator-sponsored trial of AVB-500 in combination with durvalumab in patients with platinum resistant, recurrent epithelial ovarian cancer.

Leap Therapeutics, Inc. LPTX: Additional data from the Phase 2 study of DKN-01 and Paclitaxel chemotherapy medication in endometrial cancer

Earnings

Celsion Corporation CLSN

IPOs

Finch Therapeutics Group, Inc, a clinical-stage microbiome therapeutics company developing a novel class of orally administered biological drugs, priced its upsized initial public offering of 7.5 million shares of common stock at $17 per share, for raising gross proceeds of $127.5 million.

The shares will begin trading on the Nasdaq under the ticker symbol FNCH.

China-based biopharma Connect Biopharma Holdings Ltd priced its IPO of 11.25 million ADSs, representing 11.25 million ordinary shares of the company, at $17 per ADS. The gross proceeds from the offering are expected to be $191.3 million.

The ADSs of the company that is developing T-cell therapies for chronic inflammatory diseases will begin trading on the Nasdaq under the ticker symbol CNTB.

Instil Bio, Inc., a clinical-stage biopharmaceutical company that develops an innovative cell therapy pipeline of autologous TIL therapies for the treatment of patients with cancer, priced its upsized initial public offering of 16 million shares of common stock at $20 per share. The gross proceeds of the offering, are expected to be $320 million.

The shares are expected to begin trading on the Nasdaq under the symbol TIL.

Related Link: Attention Biotech Investors: Mark Your Calendar For March PDUFA Dates