Take­da tees up pri­or­i­ty re­view for sto­ried CMV drug from its $62B Shire buy­out

One of the Shire drugs that Take­da spot­light­ed in its $62 bil­lion takeover is get­ting a speedy look at the FDA.

The drug, marib­avir, is be­ing po­si­tioned as the first and on­ly treat­ment for re­frac­to­ry post-trans­plant cy­tomegalovirus in­fec­tion — af­ter a wild 20-year ride that saw it fail stud­ies and get aban­doned while oth­er Big Phar­ma ef­forts al­so crashed.

For the Japan­ese phar­ma gi­ant, a suc­cess here would al­so bring much-need­ed val­i­da­tion in a cru­cial year in which it’s plot­ting six reg­u­la­to­ry fil­ings and ex­pect­ing four ap­provals. Two oth­ers have al­ready lined up at the FDA in re­cent months, in­clud­ing TAK-721 for eosinophilic esophagi­tis and mobo­cer­tinib for EGFR Ex­on20 in­ser­tion mu­ta­tion pos­i­tive non-small cell lung can­cer. A third, the dengue vac­cine can­di­date TAK-003, has been sub­mit­ted to the EMA.

First syn­the­sized at Glax­o­SmithK­line, marib­avir was ini­tial­ly li­censed to rare and in­fec­tious dis­ease play­er Vi­roPhar­ma, which took it through Phase II be­fore miss­ing the pri­ma­ry end­point in a Phase III study in 2009 — pre­vent­ing CMV in­fec­tions bet­ter than place­bo in pa­tients re­ceiv­ing bone mar­row trans­plants.

Obi Umeh

Vi­roPhar­ma end­ed up get­ting bought by Shire for $4.2 bil­lion in 2013, but not be­fore it pro­posed in­creas­ing the dose. Shire ran with the idea, giv­ing 4 to 12 times more drug in var­i­ous stud­ies, and got the Phase II re­sults they were look­ing for in 2016, which al­so earned a break­through des­ig­na­tion.

The rest is the clas­sic tale of big fish eat­ing medi­um fish eat­ing small fish.

Take­da spelled out the Phase III da­ta in a pre­sen­ta­tion ear­li­er this year. Of the 235 pa­tients who re­ceived marib­avir, 131 (or 55.7%) achieved clear­ance ver­sus 28 of 117 (or 23.9%) of those on con­ven­tion­al an­tivi­ral ther­a­pies (p<0.001). Viremia is the iso­la­tion of CMV by cul­ture and it’s the pri­ma­ry end­point of the study.

“CMV is one of the most com­mon vi­ral in­fec­tions ex­pe­ri­enced by trans­plant re­cip­i­ents, and cur­rent an­tivi­ral treat­ment op­tions are lim­it­ed, and physi­cians have to en­gage in a care­ful bal­ance of vi­ral clear­ance and side ef­fect man­age­ment that can im­pact pa­tient care and trans­plant out­comes,” said Obi Umeh, the pro­gram leader for marib­avir.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,700+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,700+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,700+ biopharma pros reading Endpoints daily — and it's free.