Night­star's drug for rare vi­sion loss ad­mit­ted to FDA's gene ther­a­py speed­way

In the lat­est show of reg­u­la­to­ry sup­port for gene ther­a­pies, Ox­ford spin­out Night­star Ther­a­peu­tics just got ac­cess to a fair­ly new “fast-lane” to speed its reti­nal gene ther­a­py to mar­ket.

The Lon­don-based com­pa­ny scored an FDA des­ig­na­tion called Re­gen­er­a­tive Med­i­cine Ad­vanced Ther­a­py, or RMAT, for its Phase III drug NSR-REP1. The ther­a­py is de­signed to treat choroi­deremia, a rare ge­net­ic con­di­tion that caus­es pro­gres­sive vi­sion loss, by cor­rect­ing mu­ta­tions in the CHM gene.

David Fel­lows

“Re­ceiv­ing RMAT des­ig­na­tion for NSR-REP1 high­lights the po­ten­tial of this gene ther­a­py to main­tain and im­prove vi­su­al acu­ity in choroi­deremia,” said Dave Fel­lows, Night­star’s CEO, in a state­ment. “This des­ig­na­tion fur­ther un­der­scores a recog­ni­tion of the se­ri­ous na­ture of choroi­deremia and the ur­gent need to de­vel­op new treat­ments for those af­fect­ed by in­her­it­ed reti­nal dis­eases that would oth­er­wise lead to blind­ness. We look for­ward to work­ing close­ly with the FDA to dis­cuss the NSR-REP1 de­vel­op­ment pro­gram and to de­ter­mine how we can ac­cel­er­ate the path­way for mak­ing NSR-REP1 avail­able to choroi­deremia pa­tients.”

RMAT in­cludes all the ben­e­fits of the fast track and break­through des­ig­na­tions, in­clud­ing ear­ly in­ter­ac­tions be­tween the FDA and spon­sors. Back when RMAT was first cre­at­ed (as part of the 21st Cen­tu­ry Cures Act), drug­mak­ers wor­ried gene ther­a­pies would be ex­clud­ed. That’s be­cause the FDA didn’t ex­plic­it­ly in­clude gene ther­a­pies in the first de­scrip­tion of the des­ig­na­tion. The agency reme­died that, how­ev­er, last No­vem­ber.

Un­like the break­through des­ig­na­tion, RMAT doesn’t re­quire ev­i­dence that the drug may of­fer a sub­stan­tial im­prove­ment over avail­able ther­a­pies. And like break­through des­ig­na­tions, get­ting an RMAT des­ig­na­tion doesn’t mean the prod­uct will be ap­proved and does not change the stan­dards for safe­ty and ef­fec­tive­ness need­ed for ap­proval.

RMAT des­ig­na­tion for NSR-REP1 was based on clin­i­cal da­ta sup­port­ing the main­te­nance and im­prove­ment of vi­su­al acu­ity from com­plet­ed Phase I/II tri­als in choroi­deremia pa­tients, the com­pa­ny said. The drug is now in Phase III tri­als.

Night­star’s stock $NITE, which first list­ed on Nas­daq last Sep­tem­ber, is up near­ly 6% on the news.

 

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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The lat­est Cin­derel­la sto­ry in on­col­o­gy ends with a sud­den rout as up­dat­ed da­ta dis­play spooks in­vestors

NextCure’s turn as the Cinderella of cancer-focused biotechs was short-lived.
Just a few days after its shares $NXTC zoomed up more than 250% on some very early stage results in a SITC abstract, a more complete analysis over the weekend spiked the hype and left investors in high dudgeon as the stock price collapsed back towards earth Monday.
The focus at NextCure is centered on NC318, an antibody that is intended to shut down the immunosuppressive Siglec-15 — or S-15 — target. After adding a small group of patients to the readout, investigators circled 2 clinical responses, a complete and partial response, along with 4 stable disease cases in non-small cell lung cancer.

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Te­va spin­out rais­es $85M in IPO; No­var­tis beefs up gener­ics unit with $440M deal

→ After Teva spinout 89bio recently announced that its IPO was being held up, the company is back in the game offering 5,304,687 shares at a price of $16 per share. The company has raised $84.9 million IPO in gross proceeds and will be listed under the ticker symbol $ETNB. BofA Securities, SVB Leerink and RBC Capital Markets are the joint book-running managers for the offering. Oppenheimer & Co is the co-manager for the offering.
→ Looking to amp up its presence in Japan’s hospitals, Novartis has struck a deal to buy out Aspen’s portfolio of generics in the world’s third largest healthcare market. The pharma giant is paying $440 million for Aspen’s Japanese subsidiary.
→ Novartis said tropifexor, a non-bile acid FXR agonist, has scored on several key biomarkers of NASH in a Phase IIb trial, including reductions in hepatic fat, alanine aminotransferase and body weight compared to a placebo at 12 weeks.

Break­through sta­tus and promise of a speedy re­view ar­rives for Op­di­vo/Yer­voy com­bi­na­tion as Bris­tol-My­ers bites at Bay­er

Its frontline and single-agent aspirations have been set back, but Bristol-Myers Squibb just took a big step forward in its efforts to apply its checkpoint inhibitor Opdivo to liver cancer. The FDA has granted breakthrough status and priority review to a combination, second-line treatment.

The designation is for Opdivo (nivolumab) in combination with Yervoy (ipilimumab),  for treating advanced hepatocellular carcinoma (HCC), the most common form of liver cancer. The PD-L1 drug was already approved as a single-agent, second-line treatment for HCC. A PDUFA date was set for March 10, 2020 — just 4 months from now.

Third time un­lucky: Lipocine's lat­est quest to mar­ket their oral testos­terone drug snubbed again by FDA

Lipocine’s latest attempt at securing approval for its oral testosterone drug has fizzled yet again.

The Utah-based drug developer on Monday said the FDA has spurned its marketing application, indicating that some efficacy data on the drug, Tlando, was not up to scratch to treat male hypogonadism, a condition characterized by low production of the hormone testosterone, which is responsible for maintaining muscle bulk, bone growth, and sexual function.

UP­DAT­ED: De­cry­ing 'ar­bi­trary and capri­cious' ac­tion, Re­genxBio sues FDA over clin­i­cal holds on gene ther­a­py

When RegenxBio disclosed that the FDA had placed a partial clinical hold on one of its lead gene therapies, execs outlined several customary next steps: continuing assessment and monitoring, delaying a related IND filing, and working with the FDA to address the matter.

As it turned out, they were planning something much less mundane. Two days after announcing the hold in its Q3 update, RegenxBio filed a lawsuit seeking to set it aside, the FDA Law Blog noted.

Roche's SMA chal­lenge to Bio­gen's Spin­raza fran­chise looms larg­er with piv­otal win

Roche has just landed a crucial advance in scoring a come-from-behind win on the spinal muscular atrophy field, giving Novartis and Biogen a run for their money.

The update was brief, but Roche said risdiplam hit the primary endpoint in the placebo-controlled pivotal SUNFISH trial, meeting the threshold for change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment. The results, which is the second, confirmatory portion of a two-part study, involved 180 patients with type 2 or 3 spinal muscular atrophy between 2 and 25 years old.

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Roche steers Gazy­va in­to a new PhI­II pro­gram af­ter com­bo shows promise in lu­pus nephri­tis study

Roche is working on putting together a late-stage study for its monoclonal antibody Gazyva in patients with severe kidney disease associated with lupus after a combination approach helped patients in a mid-stage study.

The 125-patient NOBILITY trial evaluated Gazyva, combined with standard-of-care treatment mycophenolate mofetil or mycophenolic acid and corticosteroids, versus standard treatment alone. The combo met the main goal of inducing a statistically superior complete renal response (CRR) of 40% at week 76, versus 18% in patients given standard treatment, Roche said.