Weekly Quick Hits (Philly) – Week of September 11, 2023

Bristol Myers Squibb reports positive results in pulmonary fibrosis study, and Arbutus discontinues development of its oral RNA destabilizer ASB-161 and its coronavirus combination therapy. For those stories and additional life science news in the Philadelphia Region, continue reading.

By Mark Terry | September 15, 2023

Funding, Awards and Collaborations?

UroGen Pharma Announces Inducement Grants Under Nasdaq Listing Rule?

Urogen Pharma (Princeton, NJ) announced the grants of inducement restricted stock units to 11 new employees, as well as options to one of the new employees, in connection with their employment with the company. The new team members will support the ongoing commercial launch of Jelmyto (mitomycin) for pyelocalyceal solution.

Arbutus Discontinues Development of AB-161 and AB-343

Warminster, Pa.-based Arbutus Biopharma Corporation announced pipeline updates, including dosing the first subject in the Phase Ia/Ib trial with AB-101, an oral PD-L1 inhibitor. The company also announced it was discontinuing the development of its oral RNA destabilizer, ASB-161, due to a preclinical toxicology finding not related to peripheral neuropathy. It is also discontinuing a coronavirus combination therapy that included AB-343 due to an unfavorable PK profile.

Rocket Pharmaceuticals Announces Pricing of Public Offering of Common Stock

Rocket Pharmaceuticals (Cranbury, NJ) announced the pricing of an underwritten public offering of 7,812,500 shares of its common stock at a public offering price of $16 per share, and pre-funded warrants to purchase 3,126,955 shares of common stock at a price of $15.00 per pre-funded warrant. The company expects to raise about $175 million.

Avalo to Divest AVTX-800 Series to AUG Therapeutics

Avalo Therapeutics (Wayne, Pa. and Rockville, Md.) inked a deal with AUG Therapeutics, LLC to sell its rights, title and interest in and assets related to AVTX-801 (D-galactose), AVTX-802 (D-mannose) and AVTX-803 (L-fucose). AUG is paying $150,000 upfront as well as a contingent milestone payment of $15 million for each compound if the FDA approval is for an indication other than a Rare Pediatric Disease, or up to 20% of certain payments granted to AUG upon any sale of any priority review voucher granted to AUG by the FDA.

Aclipse Therapeutics Inks License Deal with Chong Kun Dang Pharm. for Gastroparesis

Radnor, Pa.-based Aclipse Therapeutics and its subsidiary, Aclipse Two, entered an exclusive license agreement with Seoul, South Korea-based Chong Kun Dang Pharmaceutical Corporation for global rights, excluding South Korea, Indonesia and Vietnam, to develop CKD’s lobeglitazone for gastroparesis and additional indications. Gastroparesis is delayed gastric emptying of food in the absence of mechanical obstruction.

Madrigal Pharmaceuticals Reports Inducement Grant to New CEO

Madrigal Pharmaceuticals (Conshohocken, Pa.) announced it had made an inducement grant to Bill Sibold, the company’s new President and CEO, on September 11, 2023. It was awarded outside of its Amended 2015 Stock Plan under the terms of the company’s 2023 Inducement Plan, approved and adopted by the board on September 8, 2023. The grant was a one-time sign-on equity award of 50,000 time-based restricted stock units that will vest ratably over four years and a one-time sign-on award of performance-based restricted stock units (PSU) with a target of 50,000 PSU.

NRx Pharma Inks Support Deal with LifeSci Associates

NRx Pharmaceuticals, Inc. (Radnor, Pa.) inked an agreement with LS Associations, a division of LifeSci Advisors, in which LSA will provide certain consulting services to NRx, including but not limited to, arranging for the provision of NRx’s interim Financial Officer. As part of the deal, NRx appointed Richard Narido to act as interim CFO.

Penn’s June Named Winner of the 2024 Breakthrough Prize in Life Sciences

Carl June , the Richard W. Vague Professor in Immunotherapy at the Perelman School of Medicine and director of the Center for Cellular Immunotherapies at Penn Medicine’s Abramson Cancer Center won the 2024 Breakthrough Prize in Life Sciences. The award is for the development of CAR-T cell immunotherapy.

Penn Opens New Multi-Disciplinary Research Labs in One uCity Square

Wexford Science & Technology, LLC and the 美国宾夕法尼亚大学 signed a lease for new laboratory space at One uCity Square. The 115,000 square feet of space will host labs for novel vaccines, therapeutics, and engineered diagnostics research.

In the Clinic

Mineralys Therapeutics, Inc. Publishes Phase II Laroundostrat Treatment-Resistant Hypertension Results

Radnor, Pa.-based Mineralys Therapeutics published full results from the Target-HTN Phase II trial of lorundrostat in people with uncontrolled hypertension (uHTN) and treatment-resistant hypertension (rHTN). The drug is a highly selective aldosterone synthase inhibitor.

BMS’s LPA1 Antagonist Decreases Lung Function Decline in Phase II Pulmonary Fibrosis Study

百时美施贵宝 (Princeton, NJ) announced results from its Phase II trial of BMS-986278 in progressive pulmonary fibrosis (PPF). The drug is a potential first-in-class, oral, lysophosphatidic acid receptor 1 antagonist. The trial demonstrated that twice-daily doses of the drug over 26 weeks decreased the rate of decline in predicted forced vital capacity by 69% compared to placebo.

BriaCell Reports Patient Survival and Clinical Benefit in Breast Cancer Study

BriaCell Therapeutics Corp. (Philadelphia and Vancouver, BC) announced the completion of patient enrollment in its Phase II trial and also reported clinical data demonstrating substantial patient survival benefit and clinical benefit in advanced metastatic breast cancer patients. The study is evaluating Bria-IMT with immune checkpoint inhibitors.

Arbutus Discontinues Development of AB-161 and AB-343

Warminster, Pa.-based Arbutus Biopharma Corporation announced pipeline updates, including dosing the first subject in the Phase Ia/Ib trial with AB-101, an oral PD-L1 inhibitor. The company also announced it was discontinuing the development of its oral RNA destabilizer, ASB-161, due to a preclinical toxicology finding not related to peripheral neuropathy. It is also discontinuing a coronavirus combination therapy that included AB-343 due to an unfavorable PK profile.

Coeptis Provided Updates on Phase I trials of DVX201?

COEPTIS, Inc (Wexford, Pa.) provided safety and patient dosing updates from two Phase I trials of DVX201 for relapsed/refractory acute myeloma leukemia (AML) or high-risk myelodysplastic syndrome (MDS) and patients hospitalized with COVID-19. DVX201 is a novel allogeneic, unmodified natural killer (NK) cell therapy generated from pooled donor CD34+ hematopoietic stem and progenitor cells.

Ocugen Reports Positive Phase I/II Results for Retinitis Pigmentosa and Leber Congenital Amaurosis

Ocugen (Malvern, Pa.) provided an update for Retinitis Pigmentosa (RP) participated treated in the Phase I/II trial of OCU400 for RP associated with NR2E3 and Rhodopsin mutations and Leber congenital amaurosis (LCA) with mutations in the CEP290 gene. The announcement was an update on an extension of results provided by the company on April 14, 2023, and includes additional subjects from the high-dose group.

百时美施贵宝 Cuts 2 Mid-Stage and 4 Early-Stage Programs

Bristol Myers Squibb announced at its R&D Day that it would be slashing two mid-stage and four early-stage clinical programs. The two Phase II programs were a targeting heat shock protein 47 (HSP47) and a small interfering RNA (siRNA) for NASH. The early-stage programs included a CD47xCD20 bispecific program, a molecular glue for AML, a RIPK1 inhibitor and an anti-TIGIT lung cancer program.

New Products

ACC Released New Bioreactor Control and Data Platform, Lab Owl

InflexionPoint (ACC, Neptune, NJ) announced the launch of its new bioreactor control and data platform, Lab Owl . The platform, Lab Owl 3.0, optimizes nutrient control, oxygenation, and waste removal, demonstrating significantly improved processing efficiency and cell growth.

Research Roundup

Panavance Therapeutics Inc. Publishes Preclinical Pancreatic Cancer Data

Berwyn, Pa.-based Panavance Therapeutics published positive data in the Journal of Cellular and Molecular Medicine describing preclinical research on GP-2250 to treat pancreatic cancer. The drug is a highly selective yet broadly active cancer therapeutic with a unique mechanism of action that disrupts cancer’s energy metabolism, causing cancer cell death through several validated mechanisms.

CHOP Research: Curating Over 100 Genes for Leigh Syndrome Spectrum

An internal team led by researchers at Children's Hospital of Philadelphia (CHOP) characterized gene-disease relationships for more than 100 genes linked to Leigh syndrome spectrum, the most common pediatric form of mitochondrial disease. Many genes have been linked to Leigh syndrome since it was first identified in 1951, but there has not always been agreement about which genes are responsible for symptoms. The findings were published online in Annals of Neurology.

Drexel Research: In Support of Infections Contributing to Alzheimer’s Disease

Researchers with Drexel University’s College of Medicine found evidence that infections in the brain may contribute to Alzheimer’s disease. By analyzing 130 samples from the donated brains of 32 people, 16 with Alzheimer’s and 16 age-matched controls without Alzheimer’s, they found the Alzheimer’s brains had significantly different bacterial profiles compared to the age-matched controls. They believe it is possible that Alzheimer’s patients started with a healthy brain biome, but as the disease progresses, the healthy biome is replaced by a new set of microbes, potentially accelerating the disease.

On the Hill – Regulatory and Advocacy

Rocket Reaches FDA Alignment on Phase II Trial Design for Danon Disease

Rocket Pharmaceuticals (Cranbury, NJ) reached alignment with the FDA on the Phase II pivotal trial of RP-A501 for Danon Disease. Danon disease is a uniformly fatal inherited cardiomyopathy. There are no approved cures or disease-modifying therapies.

Madrigal’s NDA for Resmetirom for NASH Accepted for Priority Review

Madrigal Pharmaceuticals (Conshohocken, Pa.) reported the FDQ had accepted for Priority Review its New Drug Application (NDA) for resmetirom for adults with NASH with liver fibrosis. The PDUFA date is March 14, 2024.

People on the Move

MTTI Appoints Dr. Jerry Huang to Clinical Team

Molecular Targeting Technologies,Inc . (MTTI, West Chester, Pa.) appointed Dr. Jerry Huang, MD, PhD, as its Senior Vice President of Clinical Development. She has more than 20 years of global clinical development experience with expanding leadership responsibilities in the U.S., Europe, and Asia Pacific at Taiho Oncology, Inc. , 诺华 , BeiGene and 科文斯 .

Uvax Bio Appoints Dr. Pedro Garbes as VP, Global Medical Lead

Uvax Bio (Wilmington, Del.) appointed Pedro Garbes. MD, MSc. as Vice President, Global Medical Lead. Dr. Garbes most recently served as Senior Vice President at Gritstone bio .