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.

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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Den­mark's Gubra to col­lab­o­rate with Bay­er on pep­tides; Sam­sung and Bio­gen re­ceive FDA ap­proval for Lu­cen­tis biosim­i­lar

Danish biotech Gubra announced a research collaboration and license agreement with Bayer to develop peptide therapeutics to treat cardiorenal diseases. The collaboration will utilize Gubra’s peptide drug discovery platform to identify potential candidates.

This is not the first time Gubra has partnered with a company on peptide therapeutics — they partnered with Boehringer Ingelheim back in 2017 to create peptide therapeutics to treat obesity.

Ex-My­lan em­ploy­ee pleads guilty to in­sid­er trad­ing, il­le­gal­ly deal­ing on FDA ap­provals, earn­ings and Up­john merg­er

A former Mylan IT executive pleaded guilty Friday to an insider trading scheme where he bought and sold stock options on another executive’s advice.

Prosecutors secured the plea from Dayakar Mallu, Mylan’s former VP of global operations information technology, after uncovering the plan. Mallu collaborated with an unnamed “senior manager,” the SEC said, to trade options ahead of Mylan public announcements regarding FDA approvals, revenue reports and its merger with the Pfizer generics subsidiary Upjohn. The two subsequently shared profits.

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Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.