Biotech startup takes on Big Pharma rivals, reveals first-in-human data on ROS1 drug – Endpoints News

Biotech startup takes on Big Pharma rivals, reveals first-in-human data on ROS1 drug – Endpoints News

Nuvalent aimed to show that by leveraging structure-based drug design and some chemistry sizzling out of its Harvard founder’s lab, it can hit known cancer targets in ways no one else can. On Friday, it provided the first evidence in humans.

The biotech’s shares rose 62.45% to $35.74 on data from an early study showing that nearly half of the 21 heavily pretreated ROS1-positive non-small-cell lung cancer patients who received its lead drug, NVL-520, saw a partial response – a rate of 48%.

While early, Nuvalent, founded by Matt Shair, believes the positive data in this tough setting could pave the way for a swift regulatory journey.

These are patients who have exhausted all other options, CEO Jim Porter said News about endpoints from Barcelona, ​​where the data will be presented at a symposium. They would have taken the two therapies approved for their specific mutation – Novartis’ Xalkori and Roche’s Rozlytrek. In many cases, they have taken experimental drugs such as Pfizer’s lorlatinib (which is approved for another type of lung cancer like Lorbrena) and repotrectinib from Turning Point Therapeutics, now acquired by Bristol Myers Squibb. And there’s a good chance they’ve had chemotherapy, he added — even multiple courses.

“In a typical phase I, that type of patient population is very difficult to treat,” he said. “The disease has become heterogeneous at that point. We’re still seeing really encouraging activity in this heavily pretreated patient population, which really got us pretty excited about the drug and its potential.”

The Phase I study conducted by Nuvalent has enrolled 35 patients so far, and of 21 patients who were evaluable at the data cutoff date, 10 showed a partial response.

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It’s not just about the top line number, either, Porter said. From talking to doctors, Nuvalent concluded that off-target effects of previous ROS1 inhibitors have often led to interruptions, discontinuations or dose reductions – all of which can limit the drug’s effectiveness.

“We had heard loud and clear from doctors, if you could solve that, you could keep patients on therapy,” Porter said, and hopefully move on to earlier lines of treatment.

The biotech observed no dose-limiting toxicities, treatment-related serious side effects, treatment-emergent dizziness, or side effects that led to treatment reductions or discontinuations.

Looking at the specific properties they had in mind when developing NVL-520, Nuvalent said the drug appears to overcome the limitations of previous-generation drugs as they hoped.

The overall response rate was 78% among the nine patients with ROS1 G2032R mutations; 73% among the 11 with a history of CNS metastases; 53% among the most pretreated patients who received two or more prior ROS1 TKIs or one or more prior chemotherapy regimens; and 50% among the 18 who received lorlatinib or repotrectinib.

Asked about regulatory plans, Porter said Nuvalent needs to complete a phase II dose and talk to the FDA about it, but it intends to enroll both patients previously treated with ROS1 drugs and those who are kinase inhibitor-naïve.

He also noted that both Xalkori and Rozlytrek were approved in single-arm phase II studies.

“What’s interesting is that the FDA had communicated in the second approval that this is a relatively rare patient population, they don’t necessarily advocate for randomized trials,” he said, suggesting that the phase II data could serve as a basis for registration.

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But to get there, he also acknowledges that Nuvalent needs to follow patients much longer and collect more data over time. So far, patients have only been treated for anywhere from one to more than eight months, with a median of 3.6 months.

“That’s going to be very important to any decision that a regulator will make on any drug — it’s not just can you drive responses, but are they sustainable?” Porter said.

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