Weekly Quick Hits (Philly) – Week of October 23, 2023

iQure Raises $1.2 Million, Penn Researchers win $10 million breast cancer research funding and more life science news for the Philadelphia Region. Read on for more details.

By Mark Terry | October 27, 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 

Passage Bio Announced Inducement Grants

Passage Bio (Philadelphia) announced it had granted inducement awards to new employees. The company granted options to purchase 50,000 shares of common stock to the employees with an exercise price of $0.674 per share, equivalent to the company’s closing price of common stock on October 16, 2023.

Citius to Merge Subsidiary with TenX Keane to Form Citius Oncology

Citius Pharmaceuticals (Cranford, NJ) and TenX Keane Acquisition, a publicly traded special purpose acquisition company (SPAC) announced they had entered into a definitive agreement on October 23 for a proposed merger of TenX and Citius Pharma’s wholly owned oncology subsidiary. It will continue as a public company listed on the Nasdaq exchange and named Citius Oncology.

iQure Secures Funding from Ventura BioMed Investors and OKG Capital

iQure Pharma (Princeton, NJ) completed the first closing of its latest round of funding, bringing in $1.2 million. The round was led by Munich-based Ventura BioMed Investors and anchor early-stage med-tech investor OKG Capital. 

Penn Medicine Researchers Receive $10 Million Grant for Breast Cancer Prevention

A research group from the Perelman School of Medicine at the University of Pennsylvania and the Abramson Cancer Center received a $10 million grant from the Department of Defense to develop ways to prevent breast cancer recurrence. The team also reported the results of a Phase II trial that demonstrated for the first time the possibility of detecting and treating dormant tumor cells in breast cancer survivors.

Concentric by Ginkgo Partners with Penn State on Zoonotic Disease BioSurveillance

Ginkgo Bioworks is partnering with Penn State University to integrate its biosurveillance capabilities into the university’s research of 58 wildlife species for SARS-CoV-2 to monitor for potential spillover to humans. The deal is supported by a cooperative agreement awarded to Penn State from the U.S. Department of Agriculture Animal and Plant Health Inspection Service. Ginkgo’s biosecurity and public health unit is called Concentric by Ginkgo.

LianBio and BMS Ink Deal for Asian Markets

LianBio (Shanghai) and Bristol Myers Squibb (Princeton, NJ) entered into an agreement where BMS obtained LianBio’s exclusive rights to development and commercialize mavacamten in Mainland China, Hong Kong, Macau, Taiwan, Singapore and Thailand, in conjunction with termination of the exclusive license agreement LianBio previously entered into with MyoKardia, now a wholly owned subsidiary of BMS. BMS is paying LianBio a one-time payment of $350 million.

West Q3: Net Sales Grew 8.8% and Declares Q4 Dividend

West Pharmaceutical Services (Exton, Pa.) reported its third-quarter financial results, updated its full-year 2023 financial guidance and declared a fourth-quarter 2023 dividend. The company reported net sales of $747.4 million for the quarter, 8.8% growth. It is updating full-year 2023 adjusted-diluted EPS guidance to a new range of $7.95 to $8.00 compared to a previous range of $7.65 to $7.80. The Q4 dividend is $0.20 per share.

Sonnet BioTherapeutics Announces Pricing of $4.55 Million Underwritten Public Offering

Sonnet BioTherapeutics (Princeton, NJ) announced the pricing of an underwritten public offering of 2,843,750 shares of common stock and investor warrants to purchase up to an aggregate of 5,687,500 shares of common stock. Each share of common stock is being sold together with two investor warrants at a combined offering price of $1.60 for a total gross proceeds of about $4.5 million.

Avantor Q3: Net Sales Drop 7.3% to $1.72 Billion

Avantor (Radnor, Pa.) reported its third-quarter financials. Net sales were $1.72 billion for the quarter, a decrease of 7.3%. However, Michael Stubblefield, President and CEO, noted, “Disciplined execution, driven by the Avantor Business System, resulted in more than 110% free cash flow conversion and enabled ongoing debt paydown.”

Bristol Myers Squibb Q3: 8% Revenue Growth for In-Line Products and New Product Portfolio

Bristol Myers Squibb (Princeton, NJ) reported its third-quarter financials. Revenues were $11.0 billion for the quarter, with GAAP Earnings Per Share (EPS) of $0.93. Revenue growth for in-line products and new product portfolio was 8%. U.S. revenues dropped 4% to $8.7 billion for the quarter largely due to lower sales of Revlimid from generic competition.

Harmony Biosciences Selects Newest Recipients for its Patients at the Heart and Progress at the Heart Awards

Harmony Biosciences (Plymouth Meeting, Pa.) selected the latest round of nonprofit organizations for its Patients at the Heart and Progress at the Heart awards. These awards help fund nonprofits addressing challenges for people with sleep disorders and rare neurological diseases. Recipients include the Awareness & Patient Support Progression – Kleine-Levin Syndrome Foundation; PWS Sleep Within Reach Resources – Prader-Willi Syndrome Association; The Restful Revolution – Rare & Black; and Wake Up and Learn – Geisinger Health Foundation.

In the Clinic

AstraZeneca’s Imfinzi Demonstrated Robust Tumor Responses in 1^st-line Triple-Negative Breast Cancer

AstraZeneca US (Wilmington, Del.) presented updated results from the BEGONIA Phase Ib/II trial for the cohort of patients treated with datopotamab deruxtecan (Dato-DXd) plus Imfinzi (durvalumab) (Arm 7) that demonstrated the combination showed durable tumor responses with no new safety signals in patients with previously untreated advanced or metastatic triple-negative breast cancer (TNBC) with six months additional follow-up from the previous data cut-off. Dato-DXd is a specifically engineered TROP2-directed DXd antibody drug conjugate (ADC) being jointly developed by AstraZeneca and Daiichi Sankyo.

Janssen’s Erdafitinib-Releasing Intravesical Delivery System Shows Promise in Bladder Cancer

The Janssen Pharmaceutical Companies of Johnson & Johnson presented the first results from a Phase I trial of TAR-210, an intravesical delivery system that releases erdafitinib into the bladder in patients with non-muscle-invasive bladder cancer (NMIBC) with select FGFR alterations. The study categorizes patients into four cohorts based on their disease presentation. The data reported were on Cohort 1, patients with recurrent, Bacillus Calmette-Guerin (BCG)-unresponsive high-risk NMIBC who refused or were ineligible for radical cystectomy, and Cohort 3, patients with intermediate-risk NMIBC low-grade papillary disease left in situ as tumor marker lesions. 

Janssen Announced Final Analysis of Phase III Prostate Cancer Study

The Janssen Pharmaceutical Companies of Johnson & Johnson announced results from the final analysis of the Phase III MAGNITUDE trial. A pre-planned multivariate analysis showed niraparib, a PARP inhibitor, combined with abiraterone acetate and given with prednisone, improved overall survival (OS) and time to symptomatic progression (TSP) and showed a favorable trend in time to cytotoxic chemotherapy (TCC) in patients with metastatic castration-resistant prostate cancer (mCRPC) with BRCA alterations.

AstraZeneca’s Imfinzi Reduced Disease Progression or Death in Endometrial Cancer

AstraZeneca presented positive results from the primary analysis of the DUO-E Phase III trial. The study showed that Imfinzi (durvalumab) plus platinum-based chemotherapy, followed by either Imfinzi alone or Imfinzi plus Lynparza (olaparib) demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to chemotherapy alone in the overall trial population of patients with newly diagnosed advanced or recurrent endometrial cancer.

Immunocore Presented 3-Year OS Data from Uveal Cancer Trial

Immunocore (Oxfordshire, England; Conshohocken, Pa.; Rockville, Md.) announced three-year overall survival (OS) data from the KIMMTRAK (tebentafusp-tebn) Phase III trial in previously untreated HLA-A*02:01 positive patients with metastatic uveal melanoma. The long-term OS results further confirmed KIMMTRAK’s reputation as first-line standard of care for this patient population.

PDS Biotech Announces Preliminary Biomarker Study Results

PDS Biotechnology Corporation (Princeton, NJ) announced immune response data from a preliminary analysis of a subset of patients in the Phase II VERSATILE-002 trial of PDS0101 in combination with Merck’s anti-PD-1 checkpoint inhibitor Keytruda (pembrolizumab) in patients with human papillomavirus (HPV)16-positive recurrent or metastatic head and neck squamous cell carcinoma (HNSCC). The drug combination appears to lead to changes toward a TH1 predominant cytokine profile believed to be linked with improved killer T cell activity.

Elucida Presents Encouraging Solid Tumor Data

Elucida Oncology (Monmouth Junction, NJ) presented new data from its Phase I/II trial of ELU001, its lead C-Dot drug conjugate (CDC) clinical candidate. In total, 42 patients with advanced malignancies and no meaningful life-prolonging therapy options received the drug, with 34 completing at least one treatment cycle. The data suggested anti-tumor activity across all levels of FRalpha antigen expression.

Acasti Dosed 1^st Patient in Aneurysmal Subarachnoid Hemorrhage Trial

Acasti Pharma (Princeton, NJ) dosed the first patient in its pivotal Phase III STRIVE-ON trial at UTHealth Houston. The study is evaluating GTX-104, a novel, injectable formulation of nimodipine for aneurysmal Subarachnoid Hemorrhage (aSAH), a rare disease that results in bleeding over the surface of the brain in the subarachnoid space between the brain and the skull.

GSK’s RSV Vaccine, Arexvy, Has Potential to Help Adults 50-59

GlaxoSmithKline announced preliminary results from its Phase III trial of the immune response and safety of Arexvy, its RSV vaccine, in adults aged 50-59, including those at increased risk of RSV lower respiratory tract disease due to certain underlying medical conditions. The vaccine is approved in the U.S. for adults 60 years and older.

Globus Med Presented 5-Year Outcomes from the NuVasive Simplify Cervical Disc Study

Globus Medical (Audubon, Pa.) presented five-year outcomes from the NuVasive Simplify Cervical Disc study at the North American Spine Society (NASS) 38^th Annual Meeting. The study demonstrated positive, long-term clinical outcomes, a high overall composite success rate at 60 months, and statistical superiority to anterior cervical discectomy fusion (ACDF) procedural outcomes.

Ocean Biomed’s Partner Virion Dosed 1^st Patient in Phase Ib Hepatitis B Trial

Ocean Biomedical (Providence, RI) announced that its joint venture partner Virion Therapeutics (Philadelphia) dosed its first patients in a Phase Ib trial of its VRON-0200 immunotherapy for a functional cure for patients with chronic Hepatitis B virus (HBV) infection. VRON-0200 is a first-in-class checkpoint modifier designed to enhance and broaden a patient’s own immune response. The trial is currently enrolling patients in Hong Kong and New Zealand, with additional sites planned in the U.S. Initial data is expected in early 2024.

NRx Pharma Announces Further Alignment with FDA on Trials for Chronic Pain 

NRx Pharmaceuticals (Radnor, Pa.) announced further alignment with the FDA Division of Anesthesiology, Addiction Medicine, and Pain Medicine related to the development of NRX-101 for chronic pain. An FDA communication authorized the company to proceed with a pharmacokinetic study under the new IND file for the treatment of chronic pain

New Products

Olympus Announced 2 New Category I CPT Codes for iTind Procedure

Olympus Corporation (Center Valley, Pa.) announced that the American Medical Association (AMA) will issue two new Category I Current Procedural Terminology (CPT) codes for the iTind procedure. This is a minimally invasive treatment for men with lower urinary tract symptoms (LUTS) secondary to benign prostatic hyperplasia (BPH).

Research Roundup

CHOP Research: OTC Meds Control Pain in Kids After Elbow Surgery as Well as Opioids

Researchers at Children’s Hospital of Philadelphia (CHOP) published research finding that over-the-counter medications such as ibuprofen or acetaminophen after routine elbow surgery in children were as effective for pain control as opioids. The study, led by Apurva S. Shah, MD, MBA, was launched because multiple studies have questioned the need for opioids after this common surgery, with one study finding that patients used less than 25% of the opioids prescribed.

Penn Research: Why Breakthrough Covid Infections Don’t Lead to Severe Disease

Researchers in the Perelman School of Medicine identified possible reasons for why breakthrough Covid infections – infections that occur after receiving vaccines – don’t cause severe disease. The study examined the response of B and T cells in fully vaccinated people who became infected with SARS-CoV-2 during the early part of the omicron wave. They found B and T cell responses to breakthrough infections are enhanced in the vaccinated compared to unvaccinated, which generates immunological memory to prevent more severe infection.

On the Hill – Regulatory and Advocacy 

FDA Accepts for Review AstraZeneca’s sBLA for Flumist

The FDA accepted for review AstraZeneca US (Wilmington, Del.)’s supplemental Biologics License Application (sBLA) for the approval of a self- or caregiver-administered option for Flumist Quadrivalent (influenza vaccine live, intranasal), a needle-free nasal spray. If approved, it would be the first flu vaccine available to be self-administered by eligible patients or caregivers.