Weekly Quick Hits (Greater Phillly) – Week of February 20, 2023

By Mark Terry, Alex Keown, and Sarah Ellinwood
February 23, 2023

Funding and Collaborations

Verrica Prices $32.5 Million Underwritten Common Stock Offering

West Chester, Pa.-based Verrica Pharmaceuticals announced the pricing of its underwritten offering of 750,000 shares of common stock and pre-funded warrants to acquire 4,064,815 shares of common stock. The price is $6.75 per share and the pre-funded warrants are $6.7499 per share.

Center for Breakthrough Medicines (CBM) and Virion Ink Strategic Partnership

Center for Breakthrough Medicines, a CDMO based in King of Prussia, Pa., inked a strategic partnership with Virion Therapeutics (Newark, Delaware). CBM will manufacture and partner with Virion on their checkpoint modifier clinical development programs, including the First-in-Human VRON-0200 immunotherapy for patients with chronic Hepatitis B virus (HBV) infection.

Aprea Therapeutics Announces Pricing of $5.5 Million Public Offering

Aprea Therapeutics, based in Doylestown, Pa., announced the pricing of a previously announced underwritten public offering of 1,050,000 shares of its common stock. The price is $5.25 per share. The company is focused on developing novel synthetic lethality-based cancer therapeutics targeting DNA damage response pathways.

Vallon Reports 2022 Financials

Philadelphia-based Vallon reported its financials for the year, with an emphasis on its pending merger with GRI Bio. The whole-stock transaction will result in a combined company focused on advancing GRI Bio’s NKT cell regulator pipeline for the treatment of inflammatory, fibrotic and autoimmune diseases with an early focus on Idiopathic Pulmonary Fibrosis.

Aclaris Announces Q4 and Full-Year 2022 Financials

Wayne, Pa.-based Aclaris Therapeutics reported its 2022 and Q4 financial report. On the clinical front, ATI-450-RA-202 for rheumatoid arthritis is expecting topline date in the second half of this year in a Phase IIb trial; its ATI-450-HS-201 Phase IIa trial for hidradenitis suppurativa is expecting topline data in March; and ATI-450-PsA-201 is expecting topline data for a Phase IIa trial for psoriatic arthritis by the end of the year. The company has $229.8 million in cash as of December 31, 2022, which it believes can fund operations through the end of 2025.

In the Clinic

Immunocore Announces Early Phase I Data for HIV Trial

Immunocore, with offices in Oxfordshire, England, Conshohocken, Pa., and Rockville, Md., presented the initial safety and activity data with IMC-M113V for people living with HIV. The therapy is a bispecific soluble T cell receptor (TCR) therapy built on the company’s ImmTAX technology.

Passage Bio Presents More Interim Data for GM1 Gangliosidosis

Philadelphia-based Passage bio presented updated clinical date from the Phase I/II Imagine-1 trial of PBGM01, a gene therapy for GM1 gangliosidosis (GM1). The data is being presented today at the 19^th Annual WORLDSymposium.

New Patents

NRx Pharmaceuticals Adds U.S. Patent for Bipolar Depression Drug Formulation

Radnor, Pa.-based NRx Pharmaceuticals received a U.S. patent for its lead formulation, NRX-101. The drug is a glycine site NMDA antagonist in trials for bipolar depression with acute and subacute suicidality.

Research Roundup

Temple Research: New Approach to Boosting Protein Production

Researchers at Philadelphia-based Lewis Katz School of Medicine at Temple University identified a new way to increase protein production in cells by several hundredfold. The research focuses on Exin21, a sequence of 21 nucleotides for a short peptide (Qalpha). When Exin21/Qalpha is added to the coding region of a targeted gene it can significantly ramp up the production of a specific protein molecule.

Penn and U of T Researchers Turned Marmoset Cells into Stem Cells

Researchers from the University of Pennsylvania School of Veterinary Medicine, the University of Texas at San Antonio and Texas Biomedical Research Institute have rewired marmoset blood cells to have the flexibility of stem cells. They then had the stem cells take on the characteristics of sperm precursors.

Hormone Therapy for Gender Dysphoria May Increase CV Risks

Researchers with Mercy Catholic Medical Center in Darby, Pa. presented a study at the American College of Cardiology’s Annual Scientific Session Together With the World Congress of Cardiology that found people with gender dysphoria taking hormone replacement as part of gender affirmation therapy have a significantly increased risk of serious cardiac events. Those CV risks include stroke, heart attack and pulmonary embolism.

Regulatory and Advocacy

Incyte Receives Positive Opinion in Europe for Vitiligo Therapy

Incyte Corporation, headquartered in Wilmington, Del., reported the European medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending the approval of ruxolitinib cream (Opzelura) for non-segmental vitiligo with facial involvement in adults and adolescents 12 years and older. It was based on data from two pivotal Phase III trials.