Weekly Quick Hits (Philly) – Week of September 18, 2023

Wistar Institute finds possible new treatment for triple-negative breast cancer, Penn researchers develop possible vaccine against Lyme disease and more life science news for the Philly Region.

By Mark Terry | September 22, 2023

Quick Hits is BioBuzz’s weekly round up of all the life science news you don’t want to miss, in your region of interest. Don’t miss a beat; SUBSCRIBE to our weekly Quick Hits newsletter via LinkedIn (BioHealth Capital Region, Greater Philadelphia, Research Triangle Park) or get it delivered to your inbox every week.

Funding, Awards and Collaborations 

Stuart Orkin Awarded Penn’s Elaine Redding Brinster Prize for Sickle Cell Therapy

Stuart Orkin, MD, the David G. Nathan Distinguished Professor of Pediatrics, Harvard Medical School and investigator with Howard Hughes Medical Institute, was awarded the Elaine Redding Brinster Prize in Science or Medicine by the Institute for Regenerative Medicine at the University of Pennsylvania. Dr. Orkin developed a treatment for sickle cell disease. The prize is supported by an endowment from the children of Elaine Redding Brinster, and is awarded annually to a researcher who made a major discovery with unique impact on biomedicine. Each winner receives $100,000, a commemorative medal, and an invitation to present a ceremonial lecture at the University of Pennsylvania.

Marinus Q3: RAISE Trial Enrollment on Track, Expanded Commercial Launch of Xtalmy

Marinus Pharmaceuticals (Radnor, Pa.) reported its third-quarter financials and business outlook. The company indicated RAISE trial enrollment trends had returned to anticipated levels, with topline data expected in Q1 2024. It also was continuing its commercial launch of Xtalmy (ganaxolone) with estimated net product revenue between $5 and $5.2 million for the third quarter. The company reported cash of $170 to $175 million as of September 30, 2023, enough to last into Q4 2024.

Sonnet Bio Regains Nasdaq Minimum Bid Price Compliance

Sonnet BioTherapeutics (Princeton, NJ) announced it had regained compliance with the minimum bid price requirement for the Nasdaq. The company’s common shares were required to maintain a minimum closing bid price of $1.00 or more for at least 10 consecutive business days, which it did on September 15, 2023.

Jefferson’s Hien Dang Received NCI’s Biden Cancer Moonshot Award

Hien Dang, PhD, Assistant Professor and Vice Chair for Research at Thomas Jefferson University, is the recipient of one of 11 awards for the National Cancer Institute’s Biden Cancer Moonshot Awards. The Moonshot has committed $5.4 million in the first year for 11 scholars, which will help fund five years of research. Dang’s research focus is on liver cancer.

New Products

Medunik USA’s Siklos Now Covered by 19 State Medicaid Programs

Princeton, NJ-based Medunik USA, a member of Duchesnay Pharmaceutical Group, announced that government coverage of Siklos (hydroxyurea, HU) under State Medicaid programs is now available in 19 states, five with preferred coverage (FL, TX, WI, and WY), as well as the Contract Drug List in California for pediatric patient. Siklos is the only FDA-approved HU-based treatment indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in adult and pediatric patients, two years of age and older, with sickle cell anemia with recurrent moderate to painful crises.

Research Roundup

Wistar Research: Possible New Treatment for Triple-Negative Breast Cancer

Zachary Schug, PhD, assistant professor in the Molecular and Cellular Oncogenesis Program of the Ellen and Ronald Caplan Cancer Center at The Wistar Institute published research describing a double-acting mechanism for fighting triple-negative breast cancer (TNBR). The research demonstrates that silencing the ACSS2 gene may improve existing treatments. Silencing the gene impairs TNBC metabolism while also boosting the immune system’s ability to fight it.

Penn Research: Making CAR-T Work Better

Researchers at the University of Pennsylvania School of Engineering and Applied Science published research that suggested an approach to help CAR0T therapies work better while minimizing severe side effects. The researchers incorporated a sugar molecule onto the surface of CAR-T cells, which are then used as a reactive handle to create a biomaterial around the coating directly in the body. This acts as a “suit of armor” that prevents dangerous interactions with macrophages.

Penn Research: mRNA Vaccine Against Lyme Disease-Causing Bacteria

Researchers from the Perelman School of Medicine at the University of Pennsylvania proved that an experimental mRNA vaccine against infection from Borrelia burgdorferi, the bacteria that causes Lyme disease, provided protection in preclinical animal models. They also identified one of the proteins in the bacterium that elicit a potent immune response, called outer surface protein A (OspA).

Penn Research: Inflammatory Bowel Disease Linked to Atopic Dermatitis

A study out of the University of Pennsylvania School of Medicine found that adults with atopic dermatitis (AD) have a 34% greater risk of developing new-onset inflammatory bowel disease (IBD) compared to people who don’t have AD. The study also found that children have a 44% increased risk. Although the study did not focus on the cause, the researchers suspect since both diseases cause changes in the microbiome, chronic inflammation, and dysfunction in the skin and gut barrier, specific cytokine dysfunction may be involved in both.

CHOP Research: Novel Methods Using MRI to Study Diseases Modeled in Zebrafish

Zebrafish are common models for a variety of diseases, but there are challenges to evaluating organ-level defects at a microscopic level. Researchers from the Children’s Hospital of Philadelphia (CHOP) developed a noninvasive technique for using MRI in adult zebrafish. They used it to evaluate the effects of certain genetic mutations associated with mitochondrial disease.

CHOP Research: College Athletes Have Worse Post-Injury Outcomes for Concussions Outside of Sports

Researchers from the Children’s Hospital of Philadelphia (CHOP) found that college athletes had worse post-injury outcomes associated with concussions they experienced outside of sports than ones they received while playing sports. Also, female athletes with concussions outside of sports had more severe symptoms and more days in sports lost to injury compared to male athletes. The research was published in the Journal of Athletic Training.

Penn Research: Wrist Temperature Associated with Future Disease Risk

Researchers with the Perelman School of Medicine at the University of Pennsylvania published a study that suggested continuous wrist temperature monitoring provides insights into the potential for future disease risk for Type 2 diabetes, hypertension, liver disease, kidney failure, and more. The study evaluated one week of data from more than 92,000 UK Biobank participants collected at home during typical daily activities, including sleep. The findings suggest that the daily peaks and valleys in wrist temperature provided insight into up to 73 different disease conditions.

On the Hill – Regulatory and Advocacy

B. Braun Receives FDA Clearance for AQUAbase nX Reverse Osmosis System

Bethlehem, Pa.-based B. Braun Medical received 510(k) clearance from the FDA for AQUAbase nX Reverse Osmosis System. This system is a single-stage reverse osmosis device providing high-quality water for dialysis patients without the use of chemicals.

FDA Approves Orchestra BioMed’s Investigational Device Exemption for Hypertension Study

Orchestra BioMed Holdings (New Hope, Pa.) announced the FDA granted approval for an investigational device exemption (IDE) to initiate the global pivotal BACKBEAT study evaluating the efficacy and safety of atrioventricular interval modulation therapy (BackBeat CNT) for treating hypertensive patients who are indicated for a dual-chamber cardiac pacemaker. Orchestra and Medtronic inked a strategic collaboration for the development and commercialization of AVIM therapy for hypertensive pacemaker patients in July 2022.

People on the Move

CorriXR Names Deborah Moorad as New CEO

CorriXR Therapeutics (Newark, Del.) named Deoborah Moorad, MBA, as the new CEO, who will succeed co-founder Eric B. Kmiec, PhD, who has been appointed Chief Scientific Officer. Moorad previously served as CEO of Nature Technology Corp. and is an Entrepreneur-in-Residence at Cortado Ventures.

Impulse Dynamics Announces Leadership Changes

Marlton, NJ-based Impulse Dynamics announced that Jason Spees has been appointed Chief Executive Officer, Shlomi Nachman was named Chairman of the Board, and John Liddicoat, MD, was appointed to the board. Spees was previously President and Chief Commercial Officer at Impulse Dynamics. Nachman was most recently the Company Group Chairman at Johnson & Johnson, overseeing the Cardiovascular and Specialty Solutions & Vision Group. Liddicoat previously was Executive Vice President and President of the Americas Region and Enterprise Technology & Innovation function at Medtronic.

CytoSorbents Re-Appoints Kathleen Block as CFO

CytoSorbents Corporation (Princeton, NJ) re-appointed Kathleen P. Block as full-time Chief Financial Officer, effective retroactively to September 2, 2023. Block acted as CytoSorbents’ CFO for 10 years until she retired in March 2023, when she became Interim CFO as a consultant. She resumed as Interim CFO after the investigation of, and mutual termination and release agreement with, former CFO Alexander D’Amico.

Connie Collingsworth Joins Ocugen’s Business Advisory Board

Ocugen (Malvern, Pa.) announced that Connie Collingsworth will join its Business Advisory Board. Collingsworth was the former Chief Operating Officer of the Bill and Melinda Gates Foundation.