Weekly Quick Hits (BioHealth Capital Region) – Week of June 19, 2023

By Mark Terry
June 23, 2023

Funding, Awards and Collaborations

RLS and Sofie Enter Strategic Partnership for Distribution of Nuclear Medicine

RLS, Inc. (Radioisotope Life Sciences) and Virginia-based SOFIE Biosciences, a developer of PET radiopharmaceuticals, entered into a strategic partnership for RLS to distribute SOFIE nuclear medicine doses for patients. RLS will manage all patient dose preparation and distribution for SOFIE in select markets, streamlining the continuum of care for top hospitals, teaching institutions and doctors.

OpGen’s Subsidiary Curetis Receives Batch of C-Series Unyvero A30 Instruments

Rockville-based OpGen, Inc. announced its German subsidiary, Curetis GmbH, received the full set of ten Unyvero A30 C-Series instruments, which have been assembled by its development partner, DMTPe, in Germany. The instruments have successfully passed electrical and functional testing and will allow rapid sample-to-answer molecular testing with numerous chemistries and assay formats.

In the Clinic

Cartesian Therapeutics Publishes Landmark Study in The Lancet Neurology

Cartesian Therapeutics announced the publication of a landmark paper in The Lancet Neurology. The study describes Descartes-08, a cutting-edge RNA CAR-T (rCAR-T) therapy administered to patients with generalized myasthenia gravis (MG), a debilitating autoimmune neurological disease. The data demonstrate marked and long-lasting clinical improvement in patients with MG. This is the first clinical trial using rCAR-T to treat autoimmunity, and the first successful Phase II trial using an engineered cell therapy to treat autoimmunity. The results described in The Lancet Neurology suggest that rCAR-T may be useful in treating a variety of other autoimmune diseases and may overcome many of the risks and toxicities associated with conventional DNA-based CAR-T cells.

Sirnaomics Advances STP705 for Squamous Cell Carcinoma

Sirnaomics, Inc. announced the advancement of STP705 for the treatment of Squamous Cell Carcinoma in situ (isSCC) into late-stage clinical development after encouraging Phase IIa and Phase IIb clinical results were shared with the FDA in an End of Phase-II meeting. The FDA provided Sirnaomics guidance to move forward with late-stage clinical development because of the efficacious data provided as well as the widespread prevalence of Squamous Cell Carcinoma (SCC) lesions. The company said it is well-positioned to advance STP705 into a confirmatory clinical study for treatment of isSCC. Siranomics said it is “preparing to move forward in 2023 with a well-designed single dosage study as a sub-group of subjects in a large Phase III clinical study.” Sirnaomics is also studying STP705 for Basal Cell Carcinoma (BCC), which will be the next candidate to move into late-stage development pending the FDA’s review.

New Products

Cupron Introduces Cost-effective Antimicrobial Technology: Cupron Sinerji

Cupron Inc., a Virginia-based antimicrobial solutions provider, announced the significant expansion of its offerings with the introduction of Cupron Sinerji. This new adjuvant-enhanced platform offers superior antimicrobial efficacy across multiple substrates, all while maintaining an economical cost structure. Cupron Sinerji incorporates adjuvants into the copper-based formulation, resulting in a custom, flexible technology engineered to harness synergistic effects to meet a wide range of product protection and antimicrobial performance objectives. Cupron is engaged with current sales and distribution partners to make the Cupron Sinerji technology platform available to their customers. 

Research Roundup

Study: Female Babies with Congenital Diaphragmatic Hernia Have Higher Death Risk

A Johns Hopkins Children Center study using medical records from an international registry concludes that female babies with congenital diaphragmatic hernia (CDH) are slightly more likely to die within 30 to 60 days of birth than male babies with the same condition. In a report on the published The Journal of Pediatrics, investigators examine the role biological sex plays in outcomes for patients with CDH, and they found that females have about a 32% higher risk of dying than males. The researchers say it’s a small but significant difference, and that the origins are unclear but may be linked in part to overall lower birth weights for female newborns.

NextCure Presents Non-Clinical Data Defining the Mechanism of NC525 

NextCure announced preclinical data that defines the mechanism of NC525-induced apoptosis of leukemic cells, but not healthy hematopoietic cells. NC525 is a humanized monoclonal antibody (mAb) that specifically binds to LAIR-1 and is being developed for the treatment of advanced myeloid leukemias, including acute myeloid leukemia (AML), myelodysplastic syndrome and chronic myelomonocytic leukemia (CMML). High expression of LAIR-1 is seen on leukemic stem cells and blast cells, where it plays a role in survival of these cancer cells. LAIR-1 expression is relatively lower on normal hematopoietic stem cells and does not play a role in survival of normal immune cells, making it an ideal anti-leukemic target.

Regulatory and Advocacy

Precigen Receives Breakthrough Therapy Designation for PRGN-2012 AdenoVerse

Precigen announced that the FDA granted Breakthrough Therapy Designation for the first-in-class investigational PRGN-2012 AdenoVerse immunotherapy for the treatment of recurrent respiratory papillomatosis (RRP). The designation was granted based on data from a Phase I trial. PRGN-2012 induced robust de novo HPV-specific T-cell immune response in RRP patients. PRGN-2012 incorporates optimized antigen design that uses gorilla adenovector technology, part of Precigen’s proprietary AdenoVerse platform, to elicit immune responses directed against cells infected with human papillomavirus type 6 (HPV 6) or HPV type 11 (HPV 11). PRGN-2012 has previously been granted Orphan Drug Designation in patients with RRP by the FDA. 

FDA Launches Cancer Biomarker Pilot Program 

The U.S. Food and Drug Administration announced a new voluntary pilot program for certain oncology drug products used with certain corresponding in vitro diagnostic tests to help clinicians select appropriate cancer treatments for patients. Under current FDA policy, an in vitro companion diagnostic test is one that provides information essential for the safe and effective use of a corresponding treatment. In oncology, for example, specific tests may be used to identify patients, such as those with a particular genetic mutation, who may or may not benefit from certain cancer treatments. Through the pilot program, the FDA will request, from drug manufacturers, performance information for the tests used to enroll patients into the clinical trials that support drug approval.  

People on the Move

Veralox Names Jonathan Mow CEO

Frederick-based Veralox Therapeutics tapped Jonathan Mow as the company’s new chief executive officer. Mow’s appointment comes as Veralox secured $24 million in funding to advance VLX-1005 through a Phase IIa proof-of-concept study evaluating its impact on heparin-induced thromobcytopenia (HIT). Mow most recently served as CEO of PhaseBio Pharmaceuticals. Earlier in his career, Mow served as vice president, business development for Amylin Pharmaceuticals until its sale to Bristol-Myers Squibb in 2012; was co-founder and vice president, commercial and business development of Corus Pharma, Inc. until its acquisition by Gilead Sciences in 2006; and headed business development for PathoGenesis Corporation until its acquisition by Chiron Corporation in 2000. Mow also held positions at Bristol-Myers Squibb, Wyeth/Lederle International and Syntex Laboratories.

I-Mab Appoints Raj Kannan as CEO

Gaithersburg-based I-Mab, a clinical-stage immunotherapy company, announced the appointment of Raj Kannan as CEO. This appointment serves as a significant step towards further realizing I-Mab’s mission of delivering transformative therapies to patients worldwide.

Raj Kannan has more than 30 years of industry experience. He recently served as CEO of Aerie Pharmaceuticals, and before that, he held the same role  at Chiasma Pharmaceuticals.