J&J-part­nered MeiraGTx aims at Bio­gen with new eye gene ther­a­py da­ta

Sev­er­al months af­ter Bio­gen tout­ed the first proof-of-con­cept da­ta from its $800 mil­lion eye gene ther­a­py, MeiraGTx and J&J are out with their own ear­ly da­ta for a ri­val treat­ment.

In a small Phase I/II tri­al for their gene ther­a­py for the de­gen­er­a­tive eye dis­ease x-linked re­tini­tis pig­men­tosa, MeiraGTx found that the 7 pa­tients in the two low­est dose co­horts saw their vi­sion im­prove on mul­ti­ple mea­sures for 6 months. The study will pro­vide the ba­sis for the New York biotech to move in­to a piv­otal Phase III for the gene ther­a­py that J&J paid $100 mil­lion to get in on last year.

Jim List

“It’s re­al­ly ex­cit­ing da­ta,” Jim List, Janssen’s glob­al ther­a­peu­tic head for car­dio­vas­cu­lar and meta­bol­ic dis­ease, told End­points News. “This shows the po­ten­tial of the ther­a­py to have an im­prove­ment in vi­sion from this treat­ment, but al­so for this to slow or halt the pro­gres­sion of the dis­ease, and that would be amaz­ing.”

The re­tini­tis pro­gram is MeiraGTx’s lead pro­gram and one of two ma­jor gene ther­a­py plays for the dis­or­der, which is gen­er­al­ly di­ag­nosed in ado­les­cence and caus­es slow de­te­ri­o­ra­tion in vi­sion for years af­ter. Bio­gen spent $877 mil­lion last year to ac­quire Night­star Ther­a­peu­tics and their gene ther­a­py pro­gram.

In Jan­u­ary, Bio­gen pub­lished a sim­i­lar set of Phase I/II da­ta for Night­star’s treat­ment in Na­ture Med­i­cine, al­so show­ing that the drug was safe and that a sub­set of pa­tients saw im­prove­ment that was sus­tained over 3 to 6 months.

Like Bio­gen, MeiraGTx al­so found though that a sub­set of pa­tients ex­pe­ri­enced an in­flam­ma­to­ry re­sponse. Al­though that re­sponse was quick­ly re­solved with oral steroids, it ap­peared to in­ter­fere with the ef­fec­tive­ness of the drug. Pa­tients on the high­est dose of MeiraGTx’s tri­al did not see a strong im­prove­ment, and the biotech will con­duct their Phase III with the low and in­ter­me­di­ate dose.

The 4 pa­tients on the in­ter­me­di­ate dose arms saw a reti­nal sen­si­tiv­i­ty in­crease of 1.02 db, and an in­crease of cen­tral vi­su­al field pro­gres­sion rate at 1.26 db/year. Cen­tral vi­su­al field pro­gres­sion rate im­proved 1.10 db/year for the low dos­es. For the high dose, both of those num­bers were neg­a­tive.

Michel Michaelides

Michel Michaelides, lead in­ves­ti­ga­tor and MeiraGTx’s sci­en­tif­ic founder, said that Bio­gen did not look at some of the core mea­sures of vi­sion that they did. He al­so point­ed to the dif­fer­ence in im­prove­ments be­tween eyes. On­ly one of each pa­tients’ eyes re­ceived ther­a­py – but cau­tioned that cross-tri­al com­par­isons were in­her­ent­ly fraught.

“I’m not sure they have such a clear dis­par­i­ty be­tween treat­ed and un­treat­ed,” he said.

Michaelides al­so said pa­tients re­port­ed im­proved vi­sion, greater clar­i­ty, greater ease track­ing signs and read­ing on a com­put­er. There’s no place­bo-con­trol for those met­rics yet and that will have to wait for Phase II. Still, Michaelides said the ear­li­er tri­als have al­ready start­ed chang­ing sci­en­tists’ goals.

“It wasn’t long ago where the am­bi­tion for de­gen­er­a­tive dis­eases was to slow halt pro­gres­sion, and what we’re show­ing here is im­prove­ment,” he said. “That’s huge for pa­tients.”

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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David Kirn, 4D Molecular Therapeutics CEO (via website)

FDA places hold on 4D Mol­e­c­u­lar’s Fab­ry gene ther­a­py

4D Molecular Therapeutics quietly tucked an FDA clinical hold on its Fabry gene therapy into an SEC filing.

Meanwhile, the biotech issued a press release the same day after the closing bell on Thursday touting an IND for another asset, in diabetic macular edema.

The California biotech had paused enrollment of patients in its two trials of the Fabry gene therapy (4D-310) last month after three patients experienced kidney issues, all of which were resolved within four weeks. At the time, 4DMT said it would wait until the second half of this year to look at 12-month clinical data on six patients in the Phase I/II trials, one in the US and one in Taiwan and Australia.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Sanofi scraps PhI­II tri­al for Prin­cip­ia drug af­ter re­view­ing com­pe­ti­tion

Months after the FDA placed Phase III trials of Sanofi’s BTK inhibitor on hold, the company is winding down one of the studies.

Sanofi reported in its Q4 earnings that the URSA study “was discontinued after careful evaluation of the emerging competitive treatment landscape in” myasthenia gravis, a rare disease that causes muscle weakness.

The Phase III, placebo-controlled trial was testing tolebrutinib in patients with the moderate-to-severe form of the disease. It started in late 2021, according to records on clinicaltrials.gov, and was originally designed to recruit 154 participants who were receiving the standard of care.

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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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How to use ex­ter­nal con­trols: FDA spells out think­ing in new draft guid­ance

The use of real-world evidence to inform the FDA’s decision-making continues apace, with the agency releasing new draft guidance yesterday on how sponsors can compare outcomes of trial participants receiving a test treatment with outcomes in a group of people external to the trial.

The practice of externally controlled trials is common, particularly in oncology or other difficult areas where it’s not ethical or feasible to use internal controls.

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