Operation Warp Speed but for rare disease (2024)

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Good morning. Today, FDA advisers are convening to discuss Lykos Therapeutics’ MDMA-assisted psychotherapy for people with PTSD. It’s a critical meeting — MDMA could be the first Schedule I psychedelic to be deemed to have a medical use. Our reporters Olivia Goldhill and Meghana Keshavan will be live-blogging the meeting all day — tune inhere.

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The need-to-know this morning

It’s a clinical trial readout kind of morning, starting withViking Therapeuticsand its MASH treatment, called VK2809, withmid-stage results.
BridgeBio Pharmareportedlong-term resultsfrom a study of its drug called infigratinib for the treatment of achondroplasia, a form of dwarfism.
Annexon Biosciencesannounced theoutcomeof a Phase 3 study in Guillain-Barré syndrome.

AZ’s CAR-T therapy shows promise in early liver cancer trial

A next-generation CAR-T therapy from AstraZeneca substantially shrunk tumors in patients with liver cancer in a small Phase 1 trial. These data are part of abig showingby AstraZeneca at ASCO this year — the company had two plenary presentations already.

While the results of this trial — showing an overall response rate of 56.5% and a response rate of 75% at the highest dose — were impressive, it’s important to keep in mind that promising early results don’t always pan out in later-stage studies, an external researcher noted.

Novartis is flashing warning signs on its newly acquired drug

This past weekend, Novartis Chief Medical Officer Shreeram Aradhye was speaking at the company’s ASCO investor event when he was asked about pelabresib, the myelofibrosis treatment the Swiss pharma giant picked up when it acquired MorphoSys.

One might have expected Aradhye to reiterate the company’s plans to submit the drug to the Food and Drug Administration for marketing authorization. But that is definitely not what Aradhye did.

Instead, his response caught the attention of STAT’s Adam Feuerstein, who now believes the plans for pelabresib have run into serious trouble. Asked for comment, Novartis declined.

Delfi unveils new data on lung cancer test

Delfi Diagnostics announced key data yesterday about its liquid biopsy test for lung cancer: The sensitivity of its test (the frequency at which it will flag positive when cancer is actually present) is 80%, and the specificity (how often the test flags negative when no cancer is detectable) is 58%.

Delfi’s strategy is different than that of other liquid biopsy companies like Grail since it’s focusing specifically on lung cancer, which has an established mode of screening through low-dose CT scans.Delfi’s idea is to use a blood test to further stratify people who would already qualify for screening into higher-risk and lower-risk groups, motivating some to actually get the CT scans they already qualify for and allowing others to feel comfortable skipping them.

Early-stage biotech unlikely to return to ‘sugar high’ days

From STAT’s Jonathan Wosen:Areportyesterday by Ernst & Young highlights the stark contrast in market performance between early-stage and late-stage biotechs.

Publicly traded U.S. and European biotechs with experimental drugs in Phase 1 trials lost 19% of their share value between December 2021 and March 2024, while companies with assets in late-stage trials saw their shares rise more than 20%.

A parallel trend was seen in venture capital. While a select number of biopharma companies raised huge rounds from VC firms last year, including mega-rounds of$270 millionfor RNA startup Orbital and $273 for Generate Biomedicines, the number of early-stage rounds in 2023 fell by 12%.

The report notes that lower interest rates could trigger a broader resurgence in the industry, but cautions that the industry is not likely to return to its “sugar high” days of 2020 and 2021, when companies with early-stage data could easily raise heaps of money at high valuations.

The industry overall is in a more precarious financial position — the fraction of companies with less than a year’s worth of cash on hand has risen from 18% in 2021 to 31% in 2023.

However, some companies are benefiting from an unprecedented rise in private investments in public entities, orPIPEs, with deals during the first quarter of this year totaling $5.7 billion, an all-time high.

Operation Warp Speed but for rare disease

From STAT’s Jason Mast:The FDA said last week that it had selected the first participants in a program that top official Peter Marks once billed as an Operation Warp Speed for rare disease. The agency didn’t disclose which groups were picked, but a couple companies disclosed their involvement Monday morning, including Denali Therapeutics and Neurogene.

The project is technically known as “Support for clinical Trials Advancing Rare Disease Therapeutics Pilot Program,” or START — a less sexy name to distance the effort from the politics around the Covid vaccine effort and perhaps to reflect the program’s dramatically smaller scope.

Rather than massive infusions of cash, companies will receive the ability to communicate with regulators more easily, so they can get better advice as they design studies and push toward approval. To be selected, sponsors had to be in clinical trials with either a gene or cell therapy or a more conventional drug focused on neurodegeneration. Denali is developing a new enzyme replacement for Sanfilippo syndrome, a fatal neurodegenerative condition, while Neurogene is developing a gene therapy for Rett syndrome. Grace Sciences, a company developing a gene therapy for NGLY1 deficiency, an ultra-rare condition that affects the founder’s daughter, also said it was selected.