Longevity investment bulletin: CBMG, NeuroSense, D&D Pharmatech and more

The latest longevity updates from our investment news desk.

CBMG inks $245M licensing deal with Janssen

Cellular therapy company Cellular Biomedicine Group announced a global collaboration and license agreement with Janssen Biotech for two of its therapies, which have been studied for the treatment of Non-Hodgkin Lymphoma. CBMG will receive a $245 million upfront payment and milestone payments upon certain clinical development, regulatory filing, commercialization and sales achievements.  After commercialization CBMG will also receive royalties on Janssen net sales.

Under the terms of the agreement, Janssen will receive an exclusive license to develop and commercialize the therapies in geographic territories outside of China. CBMG will transfer IND and studies to Janssen and will supply the initial clinical trial materials comprised of viral vector and plasmid, as well as assist Janssen on the technology transfer. 

“We are thrilled that through this collaboration we will be able to expand the potential of these promising NHL drug candidates in the hope of helping patients worldwide,” said Tony (Bizuo) Liu, Chairman and CEO of CBMG. “We feel privileged to work with Janssen to advance the promise of our innovative medicines.”

NeuroSense reports positive results in Parkinson’s study

Neurodegenerative disease biotech NeuroSense Therapeutics (Nasdaq: NRSN) announced positive preliminary results from a novel biomarker study evaluating the potential of the company’s therapy for the treatment of Parkinson’s disease.

The study compared blood samples from 30 healthy individuals to 30 people living with PD by using neuronal derived exosomes to identify modulations in PD-associated biomarkers, including Argonaute 2 (AG02). In the study, NeuroSense observed a statistically significant decrease in levels of AGO2 in newly diagnosed PD patients when compared to the healthy control group.

“We believe these results strengthen the scientific rationale to develop NeuroSense’s platform technology for PD, as the mechanism of action may be helpful in targeting disease-specific pathways, specifically AGO2 dysregulation,” said Dr Shiran Zimri, VP of R&D at NeuroSense. “We intend to further evaluate these findings, as well as explore additional biomarkers and their involvement in the progression and pathology of PD in future studies.”

D&D announces positive results in fatty liver trial

Disease-modifying drug developer D&D Pharmatech announced positive topline results from its Phase 1 clinical trial of DD01 in patients with type 2 diabetes and non-alcoholic fatty liver disease (NAFLD). DD01 is a long-lasting, dual GLP-1/glucagon receptor agonist designed to rapidly resolve hepatic steatosis, improve glycemic control, and reduce body weight in fatty liver disease with and without co-occurring T2D and obesity.

DD01 was found to be generally safe and well tolerated. Following only four weeks of once-weekly treatment, up to 100% of patients achieved >30% liver fat reduction. A mean relative reduction in liver fat content of >50% was observed in a pooled analysis of the two high doses of DD01, whereas the change from baseline in placebo-treated subjects was <5%.

“We are thrilled to receive the positive topline results from this initial trial that show DD01 has strong potential to rapidly reduce fatty liver independent of the longer-term goal of weight loss recommended for some patients,” said Dr Adam Bell, VP of Translational Medicine and Regulatory Affairs at D&D. “Due to its rapid action and beneficial effects on glucose control, DD01 may provide an effective adjunct to diet and exercise in these patients, uncoupling the need for weight loss to precede improvements in liver health.”

Tenaya granted FDA Fast Track for heart disease gene therapy

Heart disease targeting biotech Tenaya Therapeutics (NASDAQ: TNYA) announced it has been granted FDA Fast Track designation for its gene therapy product candidate, TN-201, being developed for the treatment of hypertrophic cardiomyopathy (HCM).

TN-201 is an adeno-associated virus-based gene therapy for the treatment of HCM caused by mutations in the MYBPC3 gene, the most common genetic cause of HCM. TN-201 is designed to deliver a fully functional MYBPC3 gene to restore normal levels of myosin-binding protein, which regulates the contraction and relaxation of the heart muscle. In preclinical studies of MYBPC3 knock-out models, TN-201 has been shown to halt disease progression and demonstrated significant and durable disease reversal and survival benefit after a single dose.

“Receipt of Fast Track designation for TN-201 reflects the pressing unmet need among HCM patients whose disease is caused by MYBPC3 genetic mutations,” said Dr Whit Tingley, Chief Medical Officer of Tenaya. “As we prepare to begin dosing patients later this year, we look forward to continued close collaboration with the FDA under this designation in support of TN-201’s development.”

Cumulus EEG device gets FDA clearance

Digital health company Cumulus Neuroscience announced that its EEG headset has been granted 510(k) clearance from the US FDA. Designed for self-directed use in a patient’s home or a healthcare facility, the Cumulus EEG device is now FDA-cleared for use by adult and adolescent patients for the remote acquisition, display, and storage of electroencephalograph (EEG) data.

The headset is available in four sizes and is easily self-applied in less than five minutes. The device provides a means to initiate clinical studies remotely with data transmitted to the Cumulus Medical Device Hub, allowing healthcare professionals to review EEG recordings in real-time. This is the second medical device clearance for the headset, which previously received the UK Conformity Assessed (UKCA) mark in the United Kingdom. 

“Receiving 510(k) clearance from the FDA marks a major milestone for Cumulus, and for our biopharma innovator partners in the US and Europe who need the ability to capture clinical-grade, EEG signals from patients while at-home, to accelerate their development programs for precision CNS medicines,” said Brian Murphy, Chief Scientific Officer at Cumulus. “By providing reliable data across multiple domains of brain function, from specific disease symptoms to functional neurophysiology, clinical researchers can accelerate the development of treatments for neuropsychiatric and neurodegenerative disorders – bringing hope to millions of patients and families navigating unmet needs in the CNS ecosystem.”

Humacyte receives RMAT designation from FDA

Clinical stage biotech Humacyte (Nasdaq: HUMA) was granted a Regenerative Medicine Advanced Therapy (RMAT) designation from the US FDA for its Human Acellular Vessel (HAV) for urgent arterial repair following extremity vascular trauma. The FDA’s RMAT designation is for breakthrough therapy of regenerative medicine.

Humacyte’s HAV is a universally implantable vascular conduit for use in vascular repair. It has shown a low rate of infection in clinical trials, making it well-suited for use in settings such as vascular trauma where wounds may be contaminated with foreign material. The HAV is designed to be available off-the-shelf and ready whenever surgeons need it, potentially saving valuable time and potentially improving patient outcomes in cases requiring urgent vascular repair.

“We are delighted to receive our second RMAT designation from the Food and Drug Administration,” said Dr Cindy Cao, Chief Regulatory Officer at Humacyte. “The RMAT designation for the HAV in our lead indication of vascular trauma is an important milestone. This designation will further enhance our communication with the FDA and will provide us with a higher likelihood for an expedited review of our planned upcoming BLA filing.”

Mikra begins production of wellness-focused protein bar

Consumer longevity company Mikra Cellular Sciences said it has begun production activities on its new “Chroma” wellness-focused protein bar. The introduction of Chroma follows the recent launch of CELLF, a cellular health nutraceutical gel formulated with bioactive compounds to positively impact the brain, heart, skin and muscular systems.

“Chroma will mark Mikra’s third product but its first to offer consumers a healthy alternative nutritional bar packed with cordyceps, free of added sugars, and certified gluten-free and vegan,” said Faraaz Jamal, CEO of Mikra. “We are finalizing production activities of a truly delicious and satisfying nutritional bar that maintains a chocolatey consistency with a delightful fudge aroma. We believe that Chroma will attract a broad and diverse health conscience audience and that our grocery-focused formulation will appeal to large brick and mortar chains like Whole Foods Market, small gyms, studios and health-focused shops alike.”

Biophytis boosts management team

Age-targeting biotech Biophytis announced the appointments of Chiara Baccelli as Director of Pharmaceutical Operations and Quality Assurance and Nicolas Fellmann as Chief Financial Officer. Baccelli brings more than 20 years of experience in the development and production of pharmaceutical products at companies including UCB, Delpharm, Bioprojet and consulting firm IDD. Fellmann brings more than 30 years of experience in finance and healthcare at companies including BioAlliance Pharma, Onxéo and Pfizer.

“As we move forward to placing Sarconeos (BIO101) on market, we are pleased to strengthen our Management Committee by welcoming Chiara as Director of Pharmaceutical Operations and Quality Assurance,” said Stanislas Veillet, CEO of Biophytis. “She brings over 20 years of diverse experience in the production of pharmaceutical products in both the pharmaceutical and biotechnology industries. Nicolas, on his side, brings a strong knowledge of the pharma and biotech sectors and key skills in financing, M&A, commercial transactions and partnerships, as well as management of listed companies, we are thrilled to welcome him.”

Melio lands investment from LongeVC

Diagnostic technology company Melio secured new investment from longevity focused venture capital firm LongeVC. The company’s diagnostics solution rapidly identifies bloodborne pathogens to eliminate unnecessary prescriptions. Using AI, the platform evaluates each pathogen cell in a blood sample, and analyzes how its DNA unwinds, melts or denatures.

By accelerating the diagnostics process, Melio disrupts current inefficient pathogen testing by breaking the “one-pathogen, one-probe” paradigm in diagnostics. Instead, by eliminating the detrimental overuse of antibiotics at birth, Melio aims to deliver actionable results through its novel testing technology and encourage healthy starts and better outcomes.

“Diagnostics are an integral component of longevity care,” said Garri Zmudze, managing partner of LongeVC. “Melio’s technology makes it faster and easier for physicians to deliver personalized and effective treatments. We are thrilled to back their game-changing work.”

Rubedo announces senotherapeutic drug candidate

Longevity biopharma Rubedo Life Sciences announced the selection of its first-in-class development candidate for the treatment of dermatological conditions. Rubedo’s development candidate RLS-1496 was shown to significantly reduce senescence signatures associated with skin senescent cells in histopathological assessments.

The company’s ALEMBIC AI platform enables the detection and selective targeting of specific cell populations that drive pathology, making it possible to develop senescence signatures that uniquely identify and profile heterogeneous senescent and senescent-like cell populations in clinical samples of diseased tissues.

“We’re thrilled to nominate our first disease-modifying development candidate for our dermatology program, which targets skin senescent cell types that drive chronic degenerative dermatological conditions associated with biological aging processes,” said Marco Quarta, CEO of Rubedo Life Sciences. “Unlike current non-steroidal topical treatments that only treat the symptoms of inflammation, we believe RLS-1496 has the potential to be the first disease-modifying therapy to provide sustained relief for patients suffering from chronic conditions like dermatitis and psoriasis by targeting the skin senescent cells that cause persistent chronic inflammation.”