Renée Aguiar-Lucander

Not so fast: FDA tacks 3 more months on­to de­ci­sion date for Cal­lid­i­tas' rare kid­ney dis­or­der drug

It looks like the FDA’s de­ci­sion on Cal­lid­i­tas’ IgA nephropa­thy can­di­date Ne­fe­con is go­ing to take a few months longer than ex­pect­ed — al­low­ing time for ri­vals like Mar­tin Shkre­li’s for­mer com­pa­ny Tra­vere Ther­a­peu­tics to catch up.

Five months af­ter grant­i­ng Ne­fe­con pri­or­i­ty re­view, the FDA is ex­tend­ing its PDU­FA date from Sept. 15 to Dec. 15, Cal­lid­i­tas an­nounced on Tues­day. The Swiss biotech filed for ac­cel­er­at­ed ap­proval back in March, based on da­ta from 200 pa­tients in the first part of the Phase III Ne­fI­gArd tri­al who showed a sig­nif­i­cant re­duc­tion in pro­tein­uria, or ex­ces­sive pro­tein in the urine.

IgAN is a rare con­di­tion that dam­ages the fil­ters, or glomeruli, in­side the kid­neys, caus­ing the or­gans to lose their func­tion. This is the first time reg­u­la­tors are con­sid­er­ing an ap­proval on the ba­sis of pro­tein­uria as a sur­ro­gate end­point for ac­cel­er­at­ed ap­proval in IgAN, ac­cord­ing to Cal­lid­i­tas. High lev­els of pro­tein­uria are con­sid­ered an ear­ly sign of kid­ney func­tion de­cline.

Up­on re­view­ing the NDA, reg­u­la­tors re­quest­ed fur­ther analy­ses, in­clud­ing ad­di­tion­al in­for­ma­tion on es­ti­mat­ed glomeru­lar fil­tra­tion rate (eGFR), as fur­ther sup­port of Cal­lid­i­tas’ pro­tein­uria da­ta, the com­pa­ny said in a state­ment. The changes were clas­si­fied as ma­jor amend­ments to the NDA.

“We will con­tin­ue to co­op­er­ate close­ly with the FDA as they com­plete the re­view of our NDA,” CEO Renée Aguiar-Lu­can­der said in the state­ment.

If ap­proved, Ne­fe­con could be­come the first ap­proved treat­ment in the US for the rare kid­ney dis­or­der. But com­peti­tors like Tra­vere’s sparsen­tan and Chi­nook Ther­a­peu­tics’ atrasen­tan aren’t far be­hind.

Last month, Tra­vere (for­mer­ly Retrophin) said that IgAN pa­tients who took sparsen­tan in a piv­otal tri­al saw a mean re­duc­tion in pro­tein­uria of 49.8% from base­line at 36 weeks. That com­pares to a mean re­duc­tion of 15.1% in a group of pa­tients who took Avapro, a cur­rent stan­dard of care (p<0.0001).

While it’s dif­fi­cult to com­pare tri­als, it ap­pears as though sparsen­tan may have the up­per hand in pro­tein­uria re­duc­tion. In No­vem­ber, Cal­lid­i­tas said Ne­fe­con-treat­ed pa­tients from the first part of the Ne­fI­gArd tri­al had a 31% re­duc­tion in pro­tein­uria af­ter nine months, com­pared to a 5% re­duc­tion in those on place­bo (p=0.005).

Chi­nook is cur­rent­ly in a Phase III tri­al with atrasen­tan, an en­dothe­lin re­cep­tor an­tag­o­nist the com­pa­ny li­censed from Ab­b­Vie for an undis­closed price back in 2019. Ab­b­Vie had pre­vi­ous­ly been de­vel­op­ing atrasen­tan for di­a­bet­ic kid­ney dis­ease, but shut down a Phase III tri­al in 2017 when mon­i­tor­ing of re­nal events “re­vealed con­sid­er­ably few­er end-points than ex­pect­ed by this time.”

SVB Leerink’s Joseph Schwartz said the FDA’s de­ci­sion to ex­tend Ne­fe­con’s PDU­FA date is “not en­tire­ly shock­ing giv­en the re­cent in­creased reg­u­la­to­ry scruti­ny at the FDA.”

“While some in­vestors may view this an­nounce­ment as a neg­a­tive for TVTX and KD­NY, as both com­pa­nies are pur­su­ing sim­i­lar strate­gies for ac­cel­er­at­ed ap­proval in IgAN, we note that Ne­fe­con is a steroid which has a dis­tinct mech­a­nism from both TVTX’s sparsen­tan and KD­NY’s atrasen­tan,” he wrote to in­vestors on Tues­day.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a rather narrow market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Ex­elix­is pulls a sur­prise win in thy­roid can­cer just days ahead of fi­nal Cabome­tyx read­out

Exelixis added a thyroid cancer indication to its super-seller Cabometyx’s label on Friday — months before the FDA was expected to make a decision, and days before the company was set to unveil the final data at #ESMO21.

At a median follow-up of 10.1 months, differentiated thyroid cancer patients treated with Cabometyx (cabozantinib) lived a median of 11 months without their disease worsening, compared to just 1.9 months for patients given a placebo, Exelixis said on Monday.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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