December 4, 2023

The need-to-know this morning

Roche is paying $2.7 billion for Carmot Therapeutics, a privately held company developing treatments for obesity.
Eyepoint Pharmaceuticals saw its share price nearly quadruple in pre-market trading after its treatment for macular degeneration met its goals in a Phase 2 study.
The FDA approved Eli Lilly's Jaypirca as a treatment for chronic lymphocytic leukemia, widening its availability beyond mantle cell lymphoma.

Crispr

The backstory of sickle cell and CRISPR

For decades, sickle cell patients faced discrimination in medical settings: Most patients here are African American, and drugmakers shied away from developing treatments for this painful disease. But now, they will likely be the first to benefit from a CRISPR-based medicine: The FDA is expected to soon approve exa-cel, a sickle cell therapy made by CRISPR Therapeutics and Vertex Pharmaceuticals. The treatment showed near-curative results in clinical trials.

How did an underserved population become first in line for such a revolutionary treatment? In some ways, it is utterly rational: The genetics of the disease are straightforward and well-understood, and the disease manifests in blood cells — a part of the body that researchers can easily target. All the same, consultants recommended that CRISPR Therapeutics focus on other indications to test out its technology. That the company chose sickle cell anyway is pretty remarkable, but some advocates have voiced concern: "Are these companies just interested in sickle cell disease because they want, like a model disease, to do their initial trials and then move to other lucrative agents?" one sickle cell researcher told STAT. "Are we the guinea pigs that are being tested on to fine-tune these technologies?"

artificial intelligence

An AI drug discovery deal between AstraZeneca and Absci

AstraZeneca has signed a $247 million deal with Absci Corp. to use artificial intelligence to design cancer-fighting antibody drugs, Financial Times writes. Absci has developed AI technology that can conduct large-scale protein analysis: It measures millions of interactions between proteins, generating data that could help design novel antibodies that target cancers.

AstraZeneca plans on developing a new generation of targeted drugs that replace traditional chemotherapy.

"AI is enabling us to not only increase the success and speed of our biologics discovery process, but also enhance the diversity of the biologics we discover," one biologics R&D executive at AstraZeneca said. "AI is enabling us to not only increase the success and speed of our biologics discovery process, but also enhance the diversity of the biologics we discover."

regulatory

How an FDA policy might slow progress in pediatric cancer

A new FDA policy could prevent children with cancer from receiving much-needed treatment, opines E. Anders Kolb, president and CEO of the Leukemia and Lymphoma Society. Regulators launched an initiative called Project Optimus, which prioritizes dose optimization — that is, identifying the exact amount of a drug a patient needs to treat a disease, in order to minimize side effects. The new standards would require trial sponsors to find the minimum dose possible to be effective before a new drug goes to market.

However, this effort has only adults in mind. The new policy could delay treatment to pediatric patients, because cancer is less common in younger patients and so it can take years to enroll enough children in pediatric cancer trials. It would also make drugmakers even more reticent to run pediatric cancer trials: Children are far less financially lucrative than the millions of adults who have cancer.

"Project Optimus adds unnecessary complexity, risk, and costs, and at a point that is way too early to inform efficient drug development for children," Kolb writes. "The net effect will be that drugmakers will delay the pediatric trials or discontinue development altogether."

radiotherapy

Novartis is building a new radioligand plant in China

Novartis is pouring $85 million into a new radiotherapy production facility in China, FiercePharma writes. The company is set on expanding production of the radioligand drugs Lutathera and Pluvicto, which deliver radiation to very specific cells in the body — minimizing damage to surrounding tissues. These are delicate deliverables, however: Radioligand therapies have to reach patients within hours of being made. The plant is expected to be operational by 2026.

Novartis already has radioligand production sites in Italy, Spain, New Jersey, and Indianapolis — which, combined, are expected to produce some 250,000 doses by next year. The company is facing enormous demand for Pluvicto in particular, which is a targeted therapy for prostate cancer.

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