June 21, 2023

The need-to-know this morning

Bluebird Bio said the FDA granted priority review to its gene therapy for sickle cell disease, known as lovo-cel, with a target decision date under the Prescription Drug User Fee Act (PDUFA) of Dec. 20.

Gene therapy

Uniqure gets mixed news in Huntington's disease

A year after posting promising early results from a gene therapy for Huntington's disease, Dutch biotech Uniqure delivered a mixed update on the one-time treatment.

As STAT's Jason Mast reports, the benefits of Uniqure's treatment appeared to wane after two years among patients in the early stage trial. After one year, patients who received a low dose of the gene therapy saw their levels of mHTT, the protein that causes Huntington's, decline by about 50% from baseline on average. One year later, however, that decline was just 8% — although the patients appeared to have preserved motor function, Uniqure said, suggesting the therapy might be working.

More vexing are the data on seven patients who got a high dose of the gene therapy. Twelve months after treatment, their mHTT actually increased by about 40% on average, a result Uniqure cannot explain.

Read more.

Venture

Don't count out the humble pill

Amid dire industry warnings about the Inflation Reduction Act, which gives biologic treatments preferential treatment over small-molecule drugs, one of biotech's best-known venture capital firms is investing big in a startup devoted solely to the latter technology.

As STAT's Allison DeAngelis reports, Flagship Pioneering has put up $50 million to launch Empress Therapeutics, a company out to invent small-molecule therapies aimed a range of genetic targets with implications for immune, metabolic, and neurological diseases, as well as cancer and pain.

"Our perspective at Empress and elsewhere is that if you create something that really changes the course of disease, and really impacts a patient's life, society will value that," CEO Jason Park said. "Full stop."

Read more.

M&A

Because Lilly sure isn't

Speaking of small-molecule drugs, Eli Lilly's decision to spend $2.4 billion on DICE Therapeutics and its pipeline of oral medicines suggests the company, despite its public statements, sees a bright future for such medicines.

DICE is working on oral drugs for psoriasis and other autoimmune disorders, using the same biological mechanism behind Lilly's Taltz, an injectable drug that brings in more than $2 billion a year. The idea, analysts figure, is to invest in the medicines that might one day supplant Taltz, and it's cheaper to buy them now, in the early stages of development, than after they've demonstrated promise in later trials.

But that strategy doesn't quite jibe with what Lilly CEO David Ricks has been saying about the future of small-molecule therapies. Under the IRA, such medicines would be subject to Medicare price negotiation nine years after FDA approval, a shorter window than the 13-year period afforded to biologics. That difference is "going to really truncate investment" in small molecules, Ricks told CNBC last week, adding that "investors are already saying to me, 'Why would you invest in more small molecules when biologics get 13 years before negotiations?'"

At least in the case of DICE, he chose not to listen.

M&a

Deals are down but the dollars are steady

That's the takeaway from PwC's latest look at M&A in the life sciences, where the past 12 months have seen a substantial decline in the volume of transactions but a consistent dollar amount changing hands.

According to the report, the number of deals fell 29%, to 231, over the last year, while the total dollar value slipped just 3% to $213 billion. That's due to a handful of high-dollar transactions, including Pfizer's $45 billion acquisition of Seagen and Merck's $11 billion deal for Prometheus Biosciences.

Looking forward, PwC expects major pharmaceutical firms to remain the key drivers of M&A, both because they have the easiest access to capital and because they're facing significant patent cliffs for blockbuster medicines toward the end of this decade.

More around STAT