Selecta Revives MMA Gene Therapy Program After FDA Lifts Hold

Selecta CEO Carsten Brunn/company courtesy

Selecta CEO Carsten Brunn/courtesy Selecta Biosciences.

Selecta Biosciences is poised to move forward with development efforts for its gene therapy program for methylmalonic academia (MMA) after the U.S. Food and Drug Administration lifted the hold on its clinical trial on March 9. 

The change was highlighted alongside the announcement of positive results for the full year to December 31, 2021. The FDA sent a hold letter against the firm's Phase II/III trial of gene therapy candidate SEL-302 in November due to a lack of data on the chemistry, manufacturing process and controls related to the drug candidate. SEL-302 is a combination of ImmTOR and MMA-101. 

MMA is a rare metabolic disorder that affects the body's ability to process certain amino acids and fats, leading to metabolic acidosis, hyperammonemia and long-term complications such as developmental delays, chronic kidney disease and intellectual disability. 

"I am pleased to announce that on March 9 the FDA lifted the clinical hold on our SEL-302 gene therapy program to treat methylmalonic acidemia. We look forward to starting our phase 1 clinical trial expeditiously and to bring hope to those patients and families seeking a potentially durable and lifelong treatment for this terrible disease," commented Carsten Brunn, Ph.D., the president and chief executive officer of Selecta, in a statement. 

In January, the company released positive preclinical data on the synergistic effects of ImmTOR when combined with a Treg selective IL-2. The combined product, ImmTOR-IL, may have the capacity to improve the induction and durability of antigen-specific regulatory T cells. If development efforts succeed, it could be a first-in-class antigen-specific therapeutic. 

The good news presented at the earnings announcement was accompanied by a string of highlights and milestones for Selecta. 

These include its engineering collaboration with Cyrus Biotechnology to harness IL-2 to promote the expansion of Treg and treat patients with autoimmune illnesses, an exclusive licensing deal to use Genovis AB's IdeXork immunoglobulin G protease with ImmTOR to treat patients with pre-existing adeno-associated virus (AAV) immunity problems and a partnership with Ginkgo Bioworks to design AAV capsids and boost gene therapy delivery. Selecta also has a strategic license to develop gene therapies with Takeda Pharmaceutical, focusing on lysosomal storage disorders. 

"We are incredibly excited about our future, and we are focused on continuing to prosecute our growing wholly owned pipeline, continuing to advance our technologies to enable AAV gene therapies, supporting our numerous collaboration partners and rapidly advancing our next generation ImmTOR-IL into the clinic," Brunn added.

Selecta ended 2021 with $129.4 million in cash, cash equivalents, marketable securities and restricted cash, compared to the $140.1 million posted in the previous year. Revenue for the fourth quarter was logged at $29.9 million, from just $12 million in 2020, driven chiefly by its licensing deal with Sobi that resulted in a huge shipment of supplies and a large cost reimbursement from the Phase III DISSOLVE trial.

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