Vera’s Kidney Data Strengthen Full-Approval Case & Curium Challenges Novartis’ Lutathera

Vera's kidney drug won accelerated approval for reducing protein in the urine. Now the company reports two-year kidney-function results that address the question the FDA left open.

And Curium brings a new competitor to Lutathera, while GSK buys another development bet from Chimagen. The clinical evidence and the commercial commitments tell different stories today.

Welcome to The Pharma Closeout for Tuesday, September 15th. I'm Alex Mercer.

And I'm Maya Patel. We'll connect today's developments across the science and the business, starting with what Vera's new results actually change.

In our top story today, Vera Therapeutics has reported the final efficacy analysis from its Phase 3 kidney-disease trial. Its weekly injection, TRUTAKNA, treats adults with primary IgA nephropathy at risk of progression. The disease can progressively damage kidney function. July's accelerated approval covered reducing protein in the urine; long-term protection was still the unanswered question.

The new ORIGIN 3 results address that directly. Across two years, average kidney function was nearly stable with treatment, while the placebo group declined. That moves the evidence beyond measuring protein in the urine to whether the kidneys retain their ability to filter blood.

That addresses the clinical-benefit question left open at accelerated approval. Does the rest of the analysis point in the same direction?

It does. Vera also reports a 76% reduction in the risk of its composite kidney-disease progression outcome versus placebo. These are still sponsor-reported topline results; the detailed final presentation and publication are ahead. But the two findings support the same clinical picture.

Then Vera has a stronger clinical case to take into its full-approval filing. What do we know about safety alongside that benefit?

Vera reports broadly comparable adverse events and infections between groups. That doesn't remove the drug's warning about immunosuppression and infection risk. The full dataset still matters for judging the balance of benefit and risk.

The company plans its supplemental filing in the fourth quarter. Today's result advances that case; the FDA's full-approval decision remains ahead.

Now to our Deal and Pipeline Roundup. GSK has agreed to acquire global rights to a preclinical myeloma program from Chimagen. The potential value is up to $750 million, including milestones; the upfront payment isn't disclosed. It's a trispecific T-cell engager. GSK already has Blenrep in myeloma; this adds a different modality to that portfolio. Let's bring in Marcus Webb on the earlier relationship.

The new program is designed to engage T cells and two tumor antigens. GSK hasn't disclosed the targets and expects Phase 1 to begin in 2027. The ambition is to improve treatment in myeloma, but no human data yet show whether that design delivers better outcomes or tolerability. The transaction establishes GSK's commitment to test it.

Turning to asthma, Connect Biopharma's Phase 2 study missed its primary endpoint. It tested a single rademikibart injection during an acute attack in adults and adolescents with type-2 inflammation, alongside standard care. It didn't establish a statistically significant reduction in treatment failure over 28 days. Maya, was there a useful signal elsewhere?

There was a lung-function improvement. FEV1 measures how much air someone can forcibly breathe out in one second. At one week, the average improvement was 130 milliliters greater than placebo. That key secondary endpoint was statistically significant. It suggests an effect worth investigating, without proving the treatment prevented failure over the following month.

The company says fewer placebo patients experienced treatment failure than expected. Where does that leave its next development decision?

Connect expects results from a similarly designed COPD study later this month, before seeking FDA alignment on Phase 3. That gives it another dataset for its acute-exacerbation strategy, and a near-term decision point after this mixed asthma readout.

In a separate delivery-platform deal, Lilly is working with QurCan on genetic medicines for the central and peripheral nervous systems. The collaboration uses a nonviral delivery platform. QurCan could receive up to $237 million in milestones per program, alongside an undisclosed upfront payment and investment.

Lilly is backing a delivery platform here, rather than a named clinical asset. That could support more than one program, but neither the programs nor their number are disclosed. The agreement doesn't establish clinical delivery success, and we can't calculate an aggregate deal value from the per-program figure.

Sionna is now acting on its earlier cystic-fibrosis setback: SION-719 missed its key activity endpoint on top of standard treatment. The company won't advance that approach. A post hoc analysis excluding patients with unusual drug exposure doesn't overturn the original result.

The company is shifting toward a different combination and has announced a 46% workforce reduction. It plans to take that combination into Phase 2a in the first quarter of 2027.

At BioMarin, a different kind of development stop. The company has discontinued VOXZOGO development in children with Noonan syndrome, where it was running a Phase 2 study. BioMarin cites study feasibility and the current treatment landscape, expressly not safety or efficacy. This ends the Noonan expansion effort; it doesn't withdraw VOXZOGO's existing indication or the separate hypochondroplasia filing.

Turning to longer-term lung-cancer follow-up, AstraZeneca has reported an exploratory eight-year survival analysis from ADAURA. This is Tagrisso after surgery for EGFR-mutated non-small-cell lung cancer. In the stage two to three-A population, estimated survival at eight years was 74% with Tagrisso versus 58% with placebo.

That's a different question from whether a treatment delays recurrence soon after surgery. The long follow-up examines whether the benefit still shows up in survival years later. For an established adjuvant treatment, that durability is the news.

The estimates need their boundary attached: 127 participants didn't have additional survival follow-up and remained censored. This is an exploratory extension, not complete eight-year observation of everyone enrolled. That missing follow-up limits how confidently we can describe the late survival experience.

A separate update from Johnson and Johnson concerns first-line treatment for advanced non-small-cell lung cancer with common EGFR mutations. COPERNICUS tested subcutaneous Rybrevant Faspro alongside oral Lazcluze, with preventive measures for side effects. Does this tell us how tolerable the regimen is in practice?

It offers supportive evidence. J&J reports low rates of rash, blood clots and administration reactions in the Phase 2b study. But it was single-arm, so it can't prove the regimen safer than alternatives or isolate which preventive measure helped. The update is about treatment experience, rather than a new comparison of tumor control.

Now to Regulatory Watch. The FDA has opened applications for an expedited IND pilot under Operation TrialBlazer. It plans to select eight to ten sponsor and research-institution pairs. The program combines rolling review of application components with earlier coordination around ethics review and site activation. Applications close October 30th.

The practical question is whether those steps can move in parallel instead of waiting on one another. The FDA retains its statutory authority. A small pilot can test how much delay comes from startup coordination, but it hasn't yet demonstrated faster activation, and it doesn't promise faster approvals across the industry.

Separately, Rocket Pharmaceuticals reports FDA alignment on continued dosing of RP-A501, its gene therapy for Danon disease, a rare inherited heart condition. The company says it can proceed under a modified protocol. Its pivotal efficacy group will include twelve male patients, with dosing completion targeted for the middle of next year.

The intended accelerated-approval case will measure LAMP2 protein expression and reduction in heart-muscle mass at twelve months. Rocket reports no thrombotic microangiopathy or capillary-leak syndrome in the first three patients treated under the modified protocol. Each had at least four weeks of follow-up. That small, early observation can't settle safety.

Quick note before our second top story today — if The Pharma Closeout is how you close out your day, follow the show on Spotify or Apple Podcasts.

Curium's new radioligand entrant brings direct competition to an established neuroendocrine-tumor treatment. The company announced BEXLUTRY's FDA approval yesterday. It references Novartis' Lutathera, and Curium describes it as the first radioligand equivalent. The competitive question now extends beyond having another approved product to how it reaches the clinic.

The approved population is adults with somatostatin-receptor-positive gastroenteropancreatic neuroendocrine tumors. BEXLUTRY uses the same active radioligand, lutetium Lu 177 dotatate, as Lutathera. But the labels aren't identical in age coverage. Lutathera includes patients twelve and older; the new product is adult-only. And that description doesn't establish automatic substitution or interchangeability.

Curium says it will pair supply with site preparation, delivery and scheduling support. That fits the practical demands of radioligand care: the medicine and the treatment appointment have to come together. What can we actually tell treatment sites about that offer today?

Only that another supplier is entering, with support services promised. We can't yet say it delivers more reliable supply, wider access or a lower price. Sites still need qualified handling, radiation-safety infrastructure and the associated infusion support. Those requirements remain, whichever supplier they use.

Then the launch test is delivery: whether sites see better scheduling or supply than they get today. That would give Curium a practical point of differentiation alongside its approval.

That's today's Closeout. Vera has new evidence for the kidney-function question left open in July; Curium now has a launch to execute. Both stories have moved, and both have another test ahead.

Thanks for spending part of your day with us. Have a good evening.

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