Weekly Quick Hits (Greater Philadelphia) – Week of February 6, 2023

By Mark Terry, Alex Keown, and Sarah Ellinwood
February 10, 2023

We know, we know – you probably have a certain species of bird on your mind. 🦅 Before you scurry off to get your snacks together for Superbowl Sunday, check out some of this past week’s latest stories out of Greater Philly. 🏈

Funding

Mineralys Therapeutics Announces Pricing of Upsized IPO

Mineralys Therapeutics (Nasdaq: MLYS) announced the pricing of its upsized initial public offering of 12,000,000 shares of common stock at an initial public offering price of $16.00 per share. The gross proceeds from the offering are expected to be $192.0 million. Shares of Mineralys common stock are expected to begin trading on the Nasdaq Global Select Market today, February 10, 2023 under the ticker symbol “MLYS,” and is expected to close on February 14, 2023. 

The Innovation Space Receives $2.475 Million in Funding to Expand Facilities

The Innovation Space, based in Wilmington, Del., is receiving $2.475 million in congressional funding to upgrade and expand its scientific facilities on Wilmintgon’s Experimental Station campus. The Innovation Space expects to allocate funds toward expanded lab capabilities, including more flexible lab and office space as well as state-of-the-art biotech, chemistry and material science modular lab space.

$25 Million Gift to Penn Medicine and Children’s Hospital of Philadelphia Establishes Center for Epilepsy and Neurodevelopmental Disorders

A $25 million gift from an anonymous donor to Penn Medicine and Children’s Hospital of Philadelphia (CHOP) will establish the Center for Epilepsy and Neurodevelopmental Disorders (ENDD), accelerating collaborative research in genetic therapies for neurodevelopmental disorders. This gift will bolster the efforts of an interdisciplinary group of clinicians and scientists at Penn and CHOP, led by director Benjamin Prosser, PhD and co-directors Beverly Davidson, PhD and Ingo Helbig, MD. Dr. Prosser is an associate professor of Physiology at the University of Pennsylvania’s Perelman School of Medicine, who adjusted the focus of his research career when his own daughter, Lucy, was diagnosed shortly after birth in 2018 with a rare genetic neurodevelopmental disorder.

Children’s Hospital of Philadelphia Receives Grant from Bill & Melinda Gates Foundation to Support Mitochondrial Research

CHOP has received more than $2.3 million from the Bill & Melinda Gates Foundation. The Center for Mitochondrial and Epigenomic Medicine (CMEM) at CHOP will use the funds to help investigate if mitochondrial variation plays a role in the varying severity seen in COVID-19 infections.

M&A

Globus Medical and NuVasive to Combine to Create Global Musculoskeletal Company

Globus Medical (NYSE: GMED) and NuVasive (NASDAQ: NUVA) are entering into a definitive agreement to combine in an all-stock transaction. Under the agreement terms, NuVasive shareholders will receive 0.75 of a share of Globus Medical Class A common stock for each share of NuVasive common stock owned at the closing of the transaction. NuVasive shareholders will own approximately 28% of the combined company, and Globus Medical shareholders will own approximately 72% on a fully diluted basis.

In the Clinic

Context Therapeutics Reveals Clinical Responses from Phase 2 OATH Clinical Trial Evaluating ONA-XR for the Treatment of Endometrial Cancer

Context Therapeutics (Nasdaq: CNTX) announced that two patients have achieved a confirmed partial response (PR) among the first 12 patients (9 evaluable) enrolled in the Phase 2 OATH clinical trial evaluating Context’s oral progesterone receptor antagonist onapristone extended release (ONA-XR) in combination with anastrozole (ANA) to treat hormone receptor positive (HR+) metastatic endometrial cancer (EC).

Incyte Presents 52-Week Results from Phase 2 Study Evaluating Povorcitinib (INCB54707) in Hidradenitis Suppurativa

Incyte (Nasdaq:INCY) shared 52-week results from a Phase 2 study evaluating the efficacy and safety of oral JAK1 inhibitor povorcitinib (formerly INCB54707) in adults with hidradenitis suppurativa (HS). Data show that average efficacy was sustained for all treatment arms following the switch to povorcitinib 75 mg QD, and povorcitinib also demonstrated durable efficacy at Week 52 in high-threshold outcomes, as evidenced by 22-29% of patients achieving HS Clinical Response 100 (HiSCR100).

First Patients Enrolled in Global Phase III Studies for Bayer Investigational Oral FXIa Inhibitor

The first patients have been enrolled in the initial trials for Bayer’s OCEANIC clinical trial program, which will explore the use of asundexian (BAY2433334), an investigational oral Factor XIa (FXIa) inhibitor, in patients with atrial fibrillation (AF) and in patients with a non-cardioembolic ischemic stroke or high-risk transient ischemic attack (TIA). OCEANIC is one of the largest Phase III endeavors Bayer has initiated.

Bellerophon Therapeutics Receives IND Clearance from China NMPA for Phase III Clinical Trial Evaluating INOpulse® in Fibrotic Interstitial Lung Disease

Bellerophon Therapeutics (Nasdaq: BLPH) announced clearance of its Investigational New Drug (IND) application from the Center for Drug Evaluation of China’s National Medical Products Administration (NMPA). The study will utilize Moderate to Vigorous Physical Activity (MVPA) as the primary endpoint and be conducted in collaboration with Bellerophon’s regional partner, Baylor BioSciences.

Context’s Onapristone Demonstrates Confirmed Partial Response in Endometrial Cancer

Philadelphia-based Context Therapeutics announced that two patients hit a confirmed partial response (PR) among the first 12 patients in its Phase II OATH study. The trial is studying Context’s oral progesterone receptor antagonist onapristone extended release (ONA-XR) in combination with anastrozole for hormone receptor positive (HR+) metastatic endometrial cancer.

Century Therapeutics Doses 1^st Patient in Phase I B-Cell Lymphoma Trial

Philadelphia-headquartered Century Therapeutics dosed the first patient in the Phase I ELiPSE-1 trial of CNTY-101 in relapsed or refractory CD19-positive B-cell lymphoma. CNTY-101 is an allogeneic cell therapy product engineered with multiple complementary functionalities to improve its product profile including a CD19 CAR for tumor targeting, IL-15 support for enhanced persistence, and Allo-Evasion technology to prevent host rejection. It also has a safety switch to eliminate the drug if necessary.

Annovis Bio Receives Approval for Clinical Trial Sites in EU for Phase III Parkinson’s Study

Annovis Bio, based in Berwyn, Pa., announced additional European clinical trial sites had been approved for its ongoing Phase III study of buntanetap for Parkinson’s disease. This adds another 48 sites in five EU countries, including Italy, Spain, Hungary, Poland and Germany.  Buntanetap is an oral translational inhibitor of neurotoxic aggregating proteins (TINAPs).

Nectin Therapeutics Forges Collaboration with Merck for Anti-PVR Antibody

Nectin Therapeutics is entering into a clinical trial collaboration agreement with Merck. Nectin will evaluate the safety, tolerability, and antitumor activity of its novel anti-PVR antibody, NTX1088, in combination with KEYTRUDA® (pembrolizumab) in patients with locally advanced and metastatic solid tumors.

New Products

Center for Breakthrough Medicines Launches Precision Plasmids Manufacturing 

The Center for Breakthrough Medicines, based in King of Prussia, Pa.,  launched a new plasmid manufacturing offering to provide phase-appropriate plasmid on-demand for cell and gene therapy companies accelerating clinical development. The new platform, Precision Plasmids, are confirmed by Next Generation Sequencing (NGS) to guarantee the reproducibility of vector-based therapies that are manufactured using plasmids. 

Standard Bariatrics Awarded Bariatric Stapling Tech Deal with Premier

Standard Bariatrics, part of Teleflex, based in Wayne, Pa., inked a group purchasing organization deal with Premier for the Titan SGS powered stapling device. The device is a sterile, single patient use instrument for longitudinal transection and resection of the gastric tissue for sleve gastrectomy pouch creation.

Signant’s Novel Electronic PROMIS CAT Assessment Enhances Patient-Centric Drug Development

Signant Health now has the ability to deliver computerized adaptive tests (CATs) in clinical trials utilizing the Patient-Reported Outcome Measure Information System (PROMIS), designed to reduce patient completion burden while maintaining measurement precision of patient-reported outcomes. 

Research Roundup

CHOP Research: Identifying Risk Factors in Children with Chronic Kidney Disease

Children’s Hospital of Philadelphia (CHOP) and Nemours Children’s Health published a study that analyzed electronic health record data from PEDSnet, a national multicenter pediatric network, to identify a large cohort of children with chronic kidney disease (CKD), to evaluate CKD progression, and evaluate clinical risk factors for kidney function decline. The goal was to demonstrate that real-world EHR data can be leveraged to model kidney function decline in kids.

Wistar Researchers ID Gene Signature to Assess Cancer Risk

Wistar Institute researchers published a study in PNAS that describes a gene signature that accurately predicts the function of p53 variants. The lab monitored differences in activity in mutant and normal p53 proteins to identify any genetic markers that would indicate a p53 variant was functioning less than normal. They then used machine learning to identify the gene signature.

Princeton Research: Tumor Metabolism Slower than Expected

Researchers from Princeton Chemistry and the Ludwig Princeton Branch demonstrated an unexpected finding: that a tumor’s metabolism was slower than expected. It was generally believed that cancer cells utilize significant amounts of energy. But in five different cancer types, the research team found that tumors could proliferate on low-energy budgets partly because they neglect normal tissue functions.

Penn Research: A Cellular Pathway that Provides Innate Protection Against Ebola

Researchers at the University of Pennsylvania School of Veterinary Medicine discovered a way that human cells battle the Ebola virus’ ability to exit the infected cells. They published the research in the Proceedings of the National Academy of Sciences. A viral-host interaction stimulates host cells to increase activity of a pathway responsible for digesting and recycling proteins, known as autophagy. This allows fewer viruses from reaching the surface of the host cell.

PDS Biotech Announces Publication of Preclinical Infectimune™ Based Vaccine Studies Showing Effective Protection Against Viral Infection

PDS Biotechnology Corporation (Nasdaq: PDSB) announced that preclinical studies involving Infectimune™, PDS Biotech’s investigational immune activating platform, were published. The paper, titled, “Recombinant Protein Vaccines Formulated with Enantio-Specific Cationic Lipid R-DOTAP Induce Protective Cellular and Antibody-Mediated Immune Responses in Mice,” reported that Infectimune™ (R-DOTAP) promoted strong T cell and antibody immune responses in both single- and multi-viral antigen-containing vaccines. The vaccines stimulated antigen-specific and multi-cytokine-inducing CD8 and CD4 T cells associated with antigen-specific killing and immune memory.

On the Hill – Regulatory and Advocacy

Rocket Pharma Receives Regenerative Medicine Advanced Therapy Designation

Cranbury, NJ-based Rocket Pharmaceuticals received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for RP-A501. The therapy is the company’s AAV-based gene therapy for Danon Disease, a fatal genetic cardiac disease. The RMAT designation was based on positive data from the Phase I RP-A501 clinical trial.

People on the Move

Carisma Therapeutics Taps Moderna’s Lin Guey for SAB

Carisma Therapeutics, in Philadelphia, tapped Lin Guey, chief scientific officer of external ventures at Moderna, to its Scientific Advisory Board. Guey is a leading expert in mRNA therapeutics and oversees Moderna’s partnership with Carisma to develop in vivo CAR-M therapies. Guey brings nearly 15 years of drug development experience in program leadership, research and nonclinical development. Prior to joining Moderna, Guey served in senior leadership roles for Tessera, Xilio, Shire, and Pfizer. 

Immunome Forms New Advisory Board

Immunome, Inc. named Dr. John Lambert and Dr. Anthony Tolcher to its newly-formed board, which will prioritize selection of novel targets for antibody drug conjugates (ADCs) and T cell redirection (TCR) modalities. Lambert has been in the antibody-drug conjugate field since joining ImmunoGen in 1987, most recently serving as CSO from 2008-2015. Tolcher is a co-founder of NEXT Oncology and was previously a co-founder of South Texas Accelerated Research Therapeutics.

Orchestra BioMed Strengthens Senior Leadership Team

Orchestra BioMed Holdings added several highly accomplished industry veterans to its senior leadership team. Avi Fischer, M.D., joins as Senior Vice President, Medical Affairs and Innovation, and J.C. Simeon assumes the role of SVP, Quality. George Papandreou, Ph.D., has been promoted to General Manager and SVP of Focal Therapies (previously SVP, Quality) and Kunal Faldu has been promoted to Vice President (VP) of Pharmaceutical Development (previously Senior Director, Formulation Development).