Weekly Quick Hits (Greater Philly) – Week of March 27, 2023

By Mark Terry, Alex Keown, and Sarah Ellinwood
March 31, 2023

A special thank you to everyone who came out to our Cell & Gene Therapy Meetup last night, and a shout out to QIAGEN for sponsoring the event. We can’t wait to see you all again soon!

Funding, Awards and Collaborations

ArriVent Closes on $155 Million Series B Round

ArriVent Biopharma, headquartered in Newtown Square, Pa., announced the completion of a $155 million oversubscribed Series B financing. The funds will be used to support pivotal Phase III and additional studies of furmonertinib, a highly brain penetrant, mutant-specific EGFR kinase inhibitor.

Thomas Jefferson University Receives $15 Million Endowment from Humana

Thomas Jefferson University received an endowed and immediate use gift of $15 million from Humana to support the university’s population health efforts. $3 million will deploy data analytics and advance research to expand focused interventions to improve health disparities. The remaining $12 million endowment will establish three priority positions at the university: Humana Deanship in the College of Population Health, Humana Professorship of Community Health and Equity, and Humana Chief Community Health and Equity Officer.

In the Clinic

AstraZeneca’s Eplontersen Demonstrated Benefit in Phase III ATTRv-PN Study

AstraZeneca and Ionis reported high-level data from the NEURO-TTRansform Phase III trial of eplontersen. The drug hit its co-primary endpoints through 66 weeks in hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). With facilities in Wilmington, Del., the study was conducted in part at the Penn Presbyterian Medical Center.

NRx Pharma Safety Board Recommends Continuing on Bipolar Depression Trial

NRx Pharmaceuticals (Radnor, Pa.) indicated the independent Data Safety Monitoring Board (DSMB) reviewed the safety and efficacy data on the first 50 patients in the trial of NRX-101 for Severe Bipolar Depression and Subacute Suicidal Ideation or Behavior and recommended continued enrollment. The drug is a proprietary fixed dose combination of D-cycloserine and lurasidone.

NRG Oncology Presents Promising Molecular Targets for Cervical Cancer

NRG Oncology, a Philadelphia-based nonprofit, reported that GOG-0240, a Phase III trial, demonstrated that the incorporation of bevacizumab with chemotherapy showed a statistically significant and clinically meaningful survival benefit in recurrent and metastatic cervical carcinoma. The data suggest that ARID1A and PIK3CA could be potential targets for drug development in the cervical cancer space.

Harmony Biosciences Announces Accelerated Phase III Timeline for Idiopathic Hypersomnia

Harmony Biosciences, based in Plymouth Meeting, Pa., announced an accelerated timeline for completing its Phase II INTUNE trial of pitolisant in adults with idiopathic hypersomnia (IH). It expects to complete enrollment in the second quarter of this year with topline data expected in the fourth quarter of 2023. Short-seller Scorpion Capital is expected to file a Citizen’s Petition with the FDA to have Harmony’s Wakix (pitolisant) withdrawn for treatment of narcolepsy after reports of “alarming toxicity.”

New Products

Fujirebio Expands its Assay Menu

H.U. Group Holdings Inc. and its wholly-owned subsidiary Fujirebio, with offices in Belgium, Malvern, Pa. and Tokyo, announced the availability of the Lumipulse G NfL CSF and Lumipulse G NfL Blood assays for the fully automated random-access Lumipulse G Immunoassay systems. They allow for the quantitative measurement of Neurofilament light (NfL) in human CSF and plasma/serum within only 35 minutes.

BriaCell Plans to Spin Out Preclinical Assets into New Company

BriaCell Therapeutics, based in Philadelphia and Vancouver, British Columbia, announced plans to spin out certain preclinical pipeline assets, specifically Bria-TILsRx and PKCδ into a new company. Once the deal closes, they expect that the spinoff company will be majority-owned, controlled, and governed by BriaCell and owned by BriaCell shareholders.

Research Roundup

Wistar Research: Possible New HIV Compounds

Researchers with The Wistar Institute published research in Antimicrobial Agents and Chemotherapy that identified hopeaphenol, a natural plant-based compound, as a potential anti-HIV product. The molecule appears to block viral replication and inhibit reactivation of the “viral reservoir” that remains in the body after HIV patients receive antiretroviral therapies. 

Jefferson Research: Children with High Blood Pressure Become Adults with High Blood Pressure

Researchers at Thomas Jefferson University published research in Hypertension that found an estimated 2-5% of children have hypertension. The study found these children often became adults with high blood pressure.

Temple and Johns Hopkins Research: New Mechanisms and Treatment Possibilities for Heart Failure

Researchers at the Lewis Katz School of Medicine at Temple University and at Johns Hopkins University demonstrated that loss of a brain-derived neurotrophic factor (BDNF) underlies post-myocardial infarction (MI) heart failure. They also demonstrated in animals that BDNF levels could be replenished therapeuticly, repairing heart tissue and enabling the heart to recover.

Regulatory and Advocacy

Incyte’s Pemazyre Approved in Japan for Myeloid/Lymphoid Neoplasms

Wilmington, Del.-based Incyte announced its Pemazyre (pemigatinib) had been approved in Japan to treat myeloid/lymphoid neoplasms (MLNs) with FGFR1 fusion. The drug is a selective fibroblast growth factor receptor (FGFR) inhibitor.

Amicus’s Pombiliti Approved in Europe for Pompe Disease

Philadelphia’s Amicus Therapeutics reported the European Commission (EC) approved Pombiliti (cipaglucosidase alfa) in combination with miglustat for adults with late-onset Pompe disease. Amicus has also submitted previously requested analytical testing for miglustat, the enzyme stabilizer component of AT-GAA. Late-onset Pompe disease is a rare, neuromuscular disorder.

Chemours Announces Glycolic Acid Sustainability Program

Wilmington, Del.-based The Chemours Company announced a long-term sustainability program for its glycolic acid product portfolio at the 2023 in-cosmetics Global tradeshow. The ultimate goal of the program is to decrease the carbon footprint of all of its glycolic acid grades by 80% against a 2023 baseline.

People on the Move

Envision Appoints Jim Street to President of Technology and CTO

Philadelphia-based Envision Pharma Group appointed James Street to President of Technology, CTO and a member of the Executive Leadership Team. Prior to joining Envision, Street was Global VP at Oracle Health Sciences.

Syridex Bio’s Servance Joins Wistar’s Board of Trustees

The Wistar Institute appointed Squire Servance, founder and managing partner of the Princeton, NJ-based life sciences-focused investment firm Syridex Bio, to its Board of Trustees. Servance will serve on the Audit, Business Development and Scientific Advisory Committees.