GSK to File Jemperli in Rectal Cancer After Phase 2 Win

Celcuity just won FDA approval for a breast cancer drug that beat its trial's control arm on a hazard ratio of 0.24 — and Wall Street is already deciding which Big Pharma should buy the company.

Meanwhile AstraZeneca is paying up to $1.5 billion to buy back a drug from the spinoff it created a decade ago, Biogen's tau data finally landed at AAIC, and Roche walked away from two Huntington's programs in a single afternoon. Busy Tuesday. Let's get into it.

Welcome to The Pharma Closeout for Tuesday, July 14th. I'm Alex Mercer.

And I'm Maya Patel.

The number that matters is a hazard ratio of 0.24. This morning the FDA approved Celcuity's gedatolisib — brand name Revtorpyk — for HR-positive, HER2-negative, PIK3CA wild-type advanced breast cancer, paired with fulvestrant, with or without palbociclib. Shares closed at $111.05, up from $103.79. And Leerink is already saying out loud that this drug "belongs in the hands" of a Big Pharma.

Let's walk the data, because it's the reason people are saying that. VIKTORIA-1 — 392 patients, three arms. The triplet, gedatolisib plus fulvestrant plus palbociclib, hit a median progression-free survival of 9.3 months against 2.0 months for fulvestrant alone. That's the 0.24. The doublet came in at 7.4 months, hazard ratio 0.33. Response rate, 32% in the triplet against 1% in the control. And duration of response — 17.5 months.

Seventeen and a half months. That's not a signal, that's a franchise.

That's the word I'd use. This isn't a bridge to something else. It's durable.

So the read looks straightforward — best-in-class PI3K-pathway drug, beats Pfizer's Piqray, Celcuity is the takeout target of the week.

Here's my issue with the Piqray line. Revtorpyk's label is PIK3CA wild-type. Piqray — alpelisib — is approved for PIK3CA-mutant disease. Today, those two drugs don't compete. They sit in adjacent populations. And there's a second wrinkle nobody's pricing: this is an intravenous drug. Patients drive to an infusion center, in a market that's sprinting toward oral. So "beats Piqray" is doing work the label doesn't support yet.

That's fair — and it's exactly why the third quarter matters. Celcuity has said the supplemental filing for the PIK3CA-mutant population is coming in Q3. That's when the head-to-head actually happens. Until then they own wild-type outright, and the mutant filing is the option on top.

Which reframes the whole acquisition thesis. A buyer isn't paying for 9.3 months. They're paying to own the PI3K pathway across mutation status — wild-type now, mutant potentially by year-end, plus a prostate program in Phase 1/2. You buy the franchise before the mutant data confirms it, or you pay a lot more after.

And that's the competitive read. If you're running commercial strategy in HR-positive breast cancer, today isn't about reacting to one approval. It's about modeling what your positioning looks like when the mutant filing lands in Q3 and this stops being a niche and becomes a pathway play. The open question — does Celcuity get to launch it themselves, or does someone write the check first.

And speaking of checks being written —

Shifting to the deal side. AstraZeneca is paying $600 million upfront, up to $900 million in milestones — $1.5 billion all in, plus tiered royalties — for global rights to Dizal's Zegfrovy. That's sunvozertinib, an oral EGFR inhibitor for exon 20 insertion lung cancer. Here's the twist: AstraZeneca spun out its Chinese research hub to create Dizal roughly a decade ago. They're buying back their own pipeline. Let's bring in Marcus Webb on this one.

Also worth noting on the deal side — NextCure and Avere are merging, all-stock, with a $320 million private placement led by Fairmount and Hansoh. Lead asset is AVR-001, a once-weekly oral IL-23 receptor antagonist for psoriasis. Trades as AVRX. And the team here is the one that took Akero from pre-IPO to a significant acquisition by Novo Nordisk.

The runway is the tell. That cash funds AVR-001 through a Phase 2b psoriasis readout, a Phase 3 start, and a Phase 2b in ulcerative colitis. Oral IL-23 is a crowded corner right now. But one program across two indications is still one program. This becomes a platform the day a second mechanism reads out — not the day the merger closes.

Rapid-fire on the rest. Chai Discovery raised $400 million, tripling its valuation from seven months ago — AI discovery money is still very much on. Erasca priced a $550 million offering. And Evotec cratered 32% after a widening first-half loss and a slashed outlook. On top of that, Pfizer took an analyst downgrade, beta bumped to 0.85 from 0.78, with its meaningful catalysts — atirmociclib, the VEGF-bispecific — pushed out to 2027.

So there's your through-line for the day.

There is. The market is paying up for de-risked, already-approved assets, and punishing anyone asking it to wait. Celcuity gets rewarded for an approval. Pfizer gets marked down for a 2027 catalyst. Same appetite, two directions.

On the regulatory side — we said yesterday we'd have the first read on the AAIC tau data, so here it is. Biogen presented Phase 2 CELIA for diranersen, the Ionis-partnered tau antisense. The 60-milligram dose slowed decline on CDR-SB by 26% over 18 months, 416 patients, with CSF total tau down 50 to 65%. BioPharma Dive is calling it the clearest evidence yet that a tau drug can touch cognition.

But it missed its primary endpoint.

It did. And that's the part that matters more than the 26%. The primary was dose-response — and it failed, because the higher doses didn't beat the lower ones. Benefit was strongest at the lowest dose studied. Think about what that hands a Phase 3 team: your dose curve runs backwards, which means you can't cleanly rule out that the effect is noise. Biogen's taking it to Phase 3 anyway. So tau isn't dead — but this validates the target more than it validates this molecule.

Two Ionis stories in one segment, actually.

Right — because also on the board, Roche scrapped both of its Ionis-partnered Huntington's programs. Tominersen didn't delay progression, and the second drug went down on an animal safety signal. So one antisense target earns another trial while another washes out entirely. That's the honest state of neuro. Separately, Huyabio reported a positive Phase 3 in melanoma — HBI-8000, an HDAC inhibitor with checkpoint therapy in checkpoint-naive patients — but no efficacy magnitude yet, so I'd hold the champagne until we see the hazard ratio. And the Leqembi subcutaneous starter-dose nod we flagged yesterday is now on the FDA tracker.

So where does that leave anyone tracking the antisense platforms?

The mechanism keeps earning another trial, and the molecules keep failing the endpoints. That gap — target validated, molecule not — is exactly where the next two years of neuro value gets decided.

Looking ahead — the catalyst that outranks today's approval is Celcuity's supplemental filing for the PIK3CA-mutant population, expected in Q3. That's the one that turns a niche launch into a pathway franchise and forces the real fight with Piqray.

I'm watching the diranersen Phase 3 design — specifically which dose Biogen picks after an inverse dose-response. That single choice tells you how much they actually believe the 60-milligram signal. And AAIC runs through tomorrow, so there's more tau and amyloid data still coming out of London.

Plus the AstraZeneca–Dizal deal is targeted to close in the second half. By year-end we'll know whether Celcuity stays a standalone story or becomes an acquisition — and that one decision reshapes how everyone models the PI3K pathway.

And that is your Pharma Closeout for Tuesday, July 14th — Celcuity's Revtorpyk approval and that 0.24 hazard ratio, AstraZeneca buying Zegfrovy back from its own spinoff, and Biogen's tau data cutting both ways at AAIC. Oncology and neuro both delivered today. If you're not already subscribed, this is the week to fix that — and if this briefing saves you time, follow us on Spotify and drop a rating.

We'll be back tomorrow with the Q3 setup on Celcuity's mutant filing and the last of the AAIC data. See you then!