Precision Bio Offloads Lead Cell Therapy Program In Pivot To In Vivo Gene Editing

Precision Bio Offloads Lead Cell Therapy Program In Pivot To In Vivo Gene Editing

DNA, genomics

Precision BioSciences is in the midst of a pivotal clinical trial of an advanced cell therapy for a type of blood cancer, but that research is no longer part of its mandate. The biotech is divesting treatment rights in a deal that will expand its funding opportunities and allow it to focus on in vivo gene-editing programs.

The buyer is Imugene Ltd., which paid a $21 million advance for global rights to azercabtagene zapreleucel, or azer-cel, an allogeneic CAR T therapy for blood cancers. An Australian company will treat patients with relapsed large B-cell lymphoma (SLBCL) with a personalized cell therapy created from their own immune cells. The Imagina license includes the ability to develop three additional cancer research programs.

CAR T therapy offers a treatment option for advanced blood cancers. Currently available versions of these treatments are automated by harvesting, building and expanding the patient's immune cells in the laboratory and then injecting them into the patient. This is a long and difficult process, and many companies are trying to improve it. Precision, based in Durham, North Carolina, is one of the companies developing CAR T therapy, which is now commercially available. It is made from donor cells processed by Azer-Cell Precision's ARCUS gene editing technology. The engineering aims to prevent donor-host disease, a risky complication associated with donor cell therapy.

In May, Precision reported on 61 prospective patients in a Phase 1 dose-escalation study. The overall response rate was 83%; The complete response was 61%. In late July, Precision announced that an FDA meeting to discuss the future of the therapy had ended. Based on that discussion and Azer-Cell's clinical data, the company said it has begun discussions with "several potential strategic partners" regarding cell therapy assets.

Imugene is a cancer immunosuppressant drug available in Australia. Its portfolio includes an oncolytic virus currently in Phase 1 trials and a B-cell immunotherapy in mid-stage clinical development. The Precision contract offers a highly developed program to Australian biotech companies. In a video announcement posted on the company's website, Imugene CEO Leslie Chong said the company "will begin exploring Azer-Cell's membership beginning in 2024."

the azer-cel license makes Precision a preclinical company focused on in vivo editorial therapy. The company says the ARCUS platform uses fewer cutting enzymes than other editing technologies such as CRISPR. Its small size enables complex editing such as in vivo editing and gene insertion.

Precision's most advanced in vivo gene editing program is PGENE-HBV, a fully proprietary hepatitis B treatment in preclinical development. In regulatory filings, the company said it plans to submit an investigational application for a new drug by 2024.

It works with Novartis, for which it paid $75 million last year, to help treat inherited blood disorders such as sickle cell anemia and beta thalassemia. A separate alliance with Eli Lilly is developing an in vivo editing therapy for the treatment of Duchenne muscular dystrophy. This association has goals in liver and central nervous system diseases, but no specific disease has been identified. Two major pharmaceutical companies provide research funds for joint programs. Precision has reached an agreement with iECURE, a startup that has the rights to use ARCUS technology for four liver diseases.

Imugene's down payment is a combination of cash and principal. Under the Precision agreements, $8 million in cash and $13 million in convertible notes will be redeemed in the first year following the closing date of the transaction. The agreement also allows Precision to receive $8 million in upfront payments, as well as cash and principal. This payment is contingent upon successful completion of Phase 1b dosing in patients with relapsed large B-cell lymphoma. Authenticate is entitled to receive up to $198 million in milestone payments and royalties from sales of licensed product. For each additional program selected by Imugene, Precision is eligible to receive up to $145 million in milestone payments and sales royalties.

In its second-quarter earnings report earlier this month, Precision reported $137.8 million in cash. By spinning off its allogeneic cell therapy business, Precision expects to reduce operating costs by $20 million. The savings will stretch Precision's cash flow through the third quarter of 2025 with cash from the Imogen deal, the company said.

As we devote all our attention and resources to applying the key features of ARCUS to in vivo gene editing applications, we are poised to use ARCUS to make a significant impact on diseases of the liver and other organs. They are suitable for inserting genes or removing large defective gene sequences, Chief Executive Michael Amoroso said in a prepared statement.

Photo: iLexx, Getty Images

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