Mallinckrodt earns FDA nod for regenerative skin therapy for burns

Mallinckrodt plc’s Stratagraft gained FDA approval for use in deep partial-thickness thermal burns. The company said regenerative skin tissue therapy could lessen or eliminate the requirement of autografting of healthy skin to treat burn wounds.

Stratagraft, which includes human keratinocytes and dermal fibroblasts in an engineered, bilayer construct, is designed to mimic natural human skin, both its inner dermis-like and outer epidermis-like layers. It comprises a living dermal matrix containing dermal fibroblasts and is overlaid with human epidermal cells. Designed for topical application, it can be sutured, stapled, or secured with adhesive. Over time, the patient’s skin cells should grow to substitute the skin cells lost due to the burn. The use of Stratagraft can help avoid or reduce the amount of healthy skin required for grafting.

Peter Marks, director of FDA’s Center for Biologics Evaluation and Research, elucidated that skin grafts are often the patient’s healthy skin taken from their body and moved to the burned area to help it heal. That procedure leaves a new wound where the healthy skin was removed. This approval proffers health care professionals a novel way to treat burn wounds.

The Stratagraft BLA was based on data from the pivotal phase III study of 71 patients who had suffered 3% to 49% total body surface area complex skin defects by thermal burns for which surgical excision and autografts were indicated. Two similar areas of burn injury on the same participant were assigned either autograft or Stratagraft. Results showed that, overall, 4% of the Stratagraft-treated sites required autografting by three months. Trial investigators also demonstrated a 98% reduction in the area necessitating autograft for Stratagraft-treated sites vs. control-treated sites (p<0.0001). And the majority (83%) of Stratagraft-treated burn wounds achieved durable wound closure three months after placement.

The safety profile was comparable to autograft, with common side effects including pruritis, blisters, hypertrophic scar, and impaired healing at the treatment site. There were no reports of Stratagraft rejection in clinical studies.

RayzeBio secures USD 108 Million to boost its radiopharmaceuticals approach to solid tumors 

RayzeBio has achieved the resources to reimagine radiation in a better, more targeted approach. The San Diego-based biotech raised another USD 108 million in a Series C financing. Venrock Healthcare Capital Partners led the round along with Deerfield Management and four other new investors, as well as a list of about a dozen current backers like venBio Partners.

Radiation is a tried and true cancer treatment method, but the old concept is ripe for a refresh via radiopharmaceuticals that go inside the body and straight to the cell. RayzeBio is working on novel drug binders aimed at clinically validated solid tumor targets.

Ken Song, M.D., CEO, and the president said in a statement that the targeted radiopharmaceuticals field shows a highly clinically validated modality that has been underappreciated. The interest and confidence from their exceptional group of investors that have funded the company enable them to fully invest in their plans to be the leader in targeted radiopharmaceuticals for many cancer types.

Besides RayzeBio, Novartis is also taking part in radiation therapy. Novartis displayed strong phase 3 data at the American Society of Clinical Oncology meeting in prostate cancer. Two years after picking up the radioligand prospect in a USD 2.1 billion deal with Endocyte, Novartis disclosed that the drug could extend patients’ lives by four months and decrease the risk of death when given alongside standard of care. Novartis observes the radiopharmaceutical space as a long-term investment. 

VectorY raises USD 38 Million to develop Alzheimer’s, ALS gene therapies

VectorY Therapeutics has raised USD 38 million to fund preclinical work on gene therapies against amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease. Forbion led the seed financing in the Dutch developer of vectorized antibodies with other investors, including Eli Lilly.

AbbVie raised the profile of vectorized antibodies in 2018 and 2019 when it struck partnerships to develop treatments for Alzheimer’s and Parkinson’s diseases with Voyager Therapeutics. That partnership collapsed last year, but a small number of biotechs continue to work on the modality in the belief that it proffers a way to get antibodies to targets on the other side of the blood-brain barrier. 

Amsterdam-based VectorY joined the field last year and, having broken cover with the support of Forbion in February, has now reeled in USD 38 million in seed funding. Forbion and a leading global investment firm co-led the round with the help of BioGeneration Ventures and Lilly. 

VectorY will utilize the money to establish preclinical proof of concept for its vectorized antibodies in ALS and Alzheimer’s and set up in-house manufacturing capabilities centered on a GMP facility in the Netherlands. 

Establishing internal manufacturing capabilities will reduce VectorY’s reliance on restricted capacity at contract providers while increasing its ambition to make products quicker and at a reduced cost. The therapies in development at VectorY are based on adeno-associated virus (AAV) vectors, technology, which has been subject to capacity constraints due to the explosion of the gene therapy space.

Umoja banks USD 210 Million to bring next-gen tumor tagging immunotherapies into the clinic

Umoja Biopharma emerged last year with USD 53 million and a mission to push past the limits of immunotherapies. Now, the company is topping that haul by securing another USD 210 million to get its lead programs in bone cancer and blood cancer into the clinic. 

The funding, drawn from a laundry list of new and old investors, will also support the development of Umoja’s technology platforms and build up its manufacturing power for preclinical and clinical trials. 

The company’s pipeline is based on three immunotherapy platforms that it plans to combine in sequence eventually. 

Andy Scharenberg, M.D., CEO and co-founder of Ujoma, said in a statement that they are going to give a first medicine that will grow a cancer-fighting cell army in the body and then send that cell army telling it how to kill off the tumor. 

The first of these platforms, known as VivoVec, develops CAR-T cells inside the body to enhance the immune system. The second, TumorTag, binds to tumor cells, marking them as targets for the CAR-T cells produced by a VivoVec-based treatment. Lastly, Umoja’s RACR/CAR platform delivers FDA-approved drugs to regulate the activity of the engineered T cells within the body. 

Umoja’s lead programs comprise UB-TT170, a TumorTag-based treatment, which tags solid tumor cells that express folate receptor alpha, and UB-VV100, a CD19-targeting CAR-T treatment made inside the body. Umoja is pairing the former with a CAR-T therapy for pediatric osteosarcoma, the most common type of bone tumor in children, and the latter with a RACR molecule for the B-cell blood cancer treatment.