Weekly Quick Hits (BioHealth Capital Region) – Week of May 1, 2023

By Sarah Ellinwood
May 5, 2023

Funding, Awards and Collaborations

Cellular Biomedicine Group Partners With Janssen to Develop and Commercialize anti CD19 & CD20 Bi-Specific and anti CD20 CAR-Ts for Non-Hodgkin Lymphoma

Cellular Biomedicine Group (CBMG) has entered into a global collaboration and license agreement with Janssen Biotech (Janssen) for C-CAR039, an anti-CD19 & CD20 bi-specific chimeric antigen receptor T-cell (CAR-T) therapy, and C-CAR066, an anti-CD20 CAR-T, which have been studied for the treatment of Non-Hodgkin Lymphoma (NHL). The U.S. Food and Drug Administration (FDA) has granted C-CAR039 Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designation for relapsed/refractory Diffuse Large B-cell Lymphoma (r/r DLBCL).  Furthermore, C-CAR039 has also received Orphan Drug designation from the FDA for the treatment of Follicular lymphoma (FL). Both C-CAR039 and C-CAR066 have received FDA Investigational New Drug application clearance.

Melanoma Research Alliance Nabs $6.3 Million in Grants to Advance Melanoma Prevention, Detection and Treatment

In recognition of Melanoma Awareness Month and Melanoma Monday, the Melanoma Research Alliance (MRA), announced its new $6.3 million commitment to fund melanoma research, supporting 24 scientists at leading academic and medical institutions across the US and Europe. MRA-funded research spans the entire spectrum of the disease including melanoma prevention, diagnosis, treatment, and metastatic disease. Melanoma is the deadliest form of skin cancer and the fifth most common cancer in the United States with nearly 100,000 new cases of melanoma that will be diagnosed in the U.S. alone in 2023.

NImmune Biopharma Collaborates with NIMML Institute to Advance the Clinical Development of its LANCL Immunoregulatory Therapeutic Pipeline

NImmune Biopharma, a late-clinical-stage precision immunology biopharmaceutical company, announced the launch of a research collaboration with the NIMML Institute, a nonprofit institute dedicated to the discovery of novel immune-mediated precision medicines. The collaboration strengthens NImmune’s precision immunology capabilities by providing access to NIMML’s TITAN-X advanced computational modeling and A.I. platform for biomarker-driven clinical development. NImmune will also gain access to NIMML’s preclinical, translational, and clinical R&D and regulatory infrastructure.

Gain Therapeutics Awarded CHF 2.5 million Innosuisse Grant to Advance Lead Program in GBA1 Parkinson’s Disease

Gain Therapeutics (Nasdaq: GANX), a biotech company developing allosteric small molecule therapies, announced that Innosuisse, the Swiss Innovation Agency, has supported GT Gain Therapeutics SA with the amount of CHF 2.5 million (~$2.8 million) to develop GT-02287, Gain’s lead program for GBA1 Parkinson’s disease. The Company’s Swiss Accelerator innovation project supported by Innosuisse entitled “Harnessing Structurally Targeted Allosteric Regulators to treat Parkinson’s Disease” includes clinical pharmacology and preclinical studies with the Company’s drug candidate GT-02287, which will be conducted as the lead program progresses through Phase 1 and Phase 2 clinical studies in GBA1 Parkinson’s disease.

MaxCyte Signs Strategic Platform License with Walking Fish Therapeutics to Support the Development of B Cell Platform

MaxCyte (Nasdaq: MXCT; LSE: MXCT), a cell-engineering focused company, announced the signing of a strategic platform license (SPL) with Walking Fish Therapeutics, a biotechnology company that is rapidly advancing B cell-based therapeutics. Under the terms of the agreement, Walking Fish obtains non-exclusive clinical and commercial rights to use MaxCyte’s Flow Electroporation® technology and ExPERT™ platform. In return, MaxCyte is entitled to receive platform licensing fees, clinical milestone payments and sales-based payments.

OpGen Announces Closing of $3.5 Million Public Offering

OpGen (Nasdaq: OPGN), a precision medicine company using molecular diagnostics and bioinformatics to help combat infectious disease, announced the closing of its previously announced public offering of an aggregate of 4,495,825 shares of its common stock (or pre-funded warrants in lieu thereof) and common stock purchase warrants to purchase up to 4,495,825 shares of common stock at a combined public offering price of $0.7785 per share (or pre-funded warrant in lieu thereof) and accompanying warrant. The warrants have an exercise price of $0.7785 per share, will be exercisable beginning on the effective date of stockholder approval of the issuance of the shares upon exercise of the warrants, and will expire five years from the date of stockholder approval.

SYNAPS Dx President and CEO, Frank Amato, Named Life Sciences CEO of the Year in Maryland Tech Council 2023 ICON Awards

The Maryland Tech Council selected Amato based upon his performance as an outstanding leader and communicator, making service an integral component of the Company’s growth and profitability and demonstrated ability to take calculated risks that lead to success.

BD Recognized by DiversityInc as a Top U.S. Company for Diversity in 2023 List

BD (Becton, Dickinson and Company) (NYSE: BDX), a leading global medical technology company with a presence in Maryland, has been named a “Noteworthy Company” in DiversityInc’s annual ranking of the top U.S. companies for diversity. Since 2001, the DiversityInc Top 50 survey has been the external validator for large U.S. employers that model fairness in their talent strategy, workplace and supplier diversity practices, as well as philanthropic engagement. More than 150 employers, employing a combined total of approximately 8.3 million U.S. employees, submitted data across six key areas: leadership accountability, human capital diversity metrics, talent programs, workplace practices, supplier diversity and philanthropy.

Lantern Pharma Selects REPROCELL USA to Provide Support for the Phase 2 Harmonic™ Clinical Trial 

CRO Reprocell USA has been awarded a contract to provide support for Lantern Pharma’s Phase 2 clinical trial entitled “A Study of LP-300 With Carboplatin and Pemetrexed in Never Smokers with Advanced Lung Adenocarcinoma (HARMONIC)”. The Harmonic™ study is being conducted to determine clinical advantages for Lantern Pharma’s investigational new drug LP-300 in combination with carboplatin and pemetrexed in patients who are never smokers with lung adenocarcinoma and have relapsed after treatment with tyrosine kinase inhibitors (TKIs). 

In the Clinic

Horizon Therapeutics Initiates Phase 3 Clinical Trial in Japan Evaluating TEPEZZA® (teprotumumab-trbw) in Adults with Chronic/Low Clinical Activity Score (CAS) Thyroid Eye Disease (TED)

Horizon Therapeutics (Nasdaq: HZNP) announced that the first patient has been enrolled in a Phase 3 clinical trial in Japan evaluating TEPEZZA for the treatment of adults with chronic Thyroid Eye Disease (TED) and low clinical activity score (CAS), which is a measure of disease activity. The trial includes patients with an initial diagnosis of TED between two to 10 years prior to the study and low levels of disease activity. Findings from this study will build on a separate Phase 3 clinical trial (OPTIC-J) that is currently underway in Japan and is evaluating TEPEZZA for the treatment of TED patients with disease duration of nine months or less and higher levels of disease activity.

Aditxt Subsidiary Adimune Signs Clinical Trial Agreement with Top Research Institution to Study Stiff Person Syndrome

Aditxt®, Inc. (NASDAQ: ADTX), a global company focused on therapeutics and technologies that modulate and monitor the immune system, announced that its subsidiary Adimune™ has signed a clinical trial agreement with Mayo Clinic to advance clinical studies targeting autoimmune diseases of the central nervous system with the initial focus on the rare, but debilitating, autoimmune disease Stiff Person Syndrome. Adimune was founded earlier this year to lead Aditxt’s immune modulation therapeutic programs.

Alphyn Biologics Reports Positive Pediatric Results from Phase 2a Atopic Dermatitis Trial

Alphyn Biologics, a clinical-stage dermatology company, announced positive results from the pediatric population enrolled in its Phase 2a clinical trial of AB-101a, a novel topical candidate for mild-to-moderate atopic dermatitis (AD), at the 22nd European Society for Pediatric Dermatology Congress (ESPD). The first-of-its-kind trial met all primary and secondary efficacy endpoints in the pediatric population ages 2 and older with minimal safety and side effects.

New Products

Charles River Laboratories Launches Next Generation Sequencing Services for Bacterial Identification and Fungal ID

Charles River Laboratories International (NYSE: CRL) announced the launch of Accugenix® Next Generation Sequencing for Bacterial Identification and Fungal Identification (Accugenix® NGS). Accugenix NGS simultaneously sequences millions of individual DNA fragments from a sample and provides key information to pharmaceutical and personal care manufacturers regarding microbial control. When studying pure isolates and/or sequencing of mixed species environments, Accugenix NGS provides a deeper resolution of organism genes and variants at a level never before possible. The platform answers client questions, provides more robust compliance capabilities and higher levels of control, while offering partnership with a global network of labs, bioinformaticians, and specialists who interpret results and provide post-report support.

Regulatory and Advocacy

FDA Approves GSK’s AREXVY, the World’s First Respiratory Syncytial Virus (RSV) Vaccine for Older Adults

GSK (LSE/NYSE: GSK) announced that the US Food and Drug Administration (FDA) has approved AREXVY (respiratory syncytial virus vaccine, adjuvanted) for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in individuals 60 years of age and older. This is the first RSV vaccine for older adults to be approved anywhere in the world.

FDA Takes Additional Steps to Advance Decentralized Clinical Trials

The U.S. Food and Drug Administration is taking additional steps to support the use of decentralized clinical trials (DCTs) for drugs, biologics and devices, where some or all the trial-related activities occur at locations other than traditional clinical trial sites. This week the agency released a new draft guidance that provides recommendations for sponsors, investigators and other stakeholders regarding the implementation of DCTs to advance medical product development and research. Examples of decentralized elements include obtaining laboratory tests at a local facility rather than a research medical center or conducting a clinical follow-up visit in the trial participant’s home using telemedicine.

Vanda Pharmaceuticals Sues Federal Government for Misappropriation of Trade Secrets and Confidential Information

Vanda Pharmaceuticals (Nasdaq: VNDA) announced its action against the federal government for the uncompensated taking and misuse of Vanda’s trade secrets and confidential information. Vanda alleges that the Food and Drug Administration improperly provided confidential details from Vanda’s drug master files for its products, HETLIOZ® and Fanapt®, to generic drug manufacturers during the FDA’s review of the manufacturers’ Abbreviated New Drug Applications. The action was filed in the United States Court of Federal Claims and seeks an award of economic damages. (Vanda Pharmaceuticals Inc. v. USA, case no. 1:23-cv-00629-AOB).

AACC Releases Guidance on Procalcitonin Testing to Improve Care for Patients with Bacterial Infections

AACC has issued expert guidance on clinical testing for procalcitonin, a blood marker that helps detect serious bacterial infections and sepsis. By giving clinicians and laboratory professionals much-needed clarity on how to best use procalcitonin tests, the new guidelines could improve the treatment of critically ill patients and those with certain lower respiratory infections.