Hey, wait a sec­ond. What about Roche’s PhI­II he­mo­phil­ia A drug?

Large­ly over­shad­owed by Bio­Marin’s po­ten­tial­ly land­mark gene ther­a­py re­sults for he­mo­phil­ia A, a group from Roche’s Chugai sub­sidiary turned up at the same sci­en­tif­ic con­fer­ence this week to spot­light up­dat­ed ev­i­dence that their bis­pe­cif­ic an­ti­body emi­cizum­ab dra­mat­i­cal­ly slashed the rate of bleed­ing episodes in a small but sig­nif­i­cant clin­i­cal tri­al.

Emi­cizum­ab, for­mer­ly ACE910, has been re­peat­ed­ly tapped by Roche’s Daniel O’Day as a top block­buster can­di­date for the phar­ma gi­ant. But now the in­jectable will have to com­pete for at­ten­tion with Bio­Marin and ri­vals who are work­ing with once-and-done gene ther­a­pies that promise to fix the bi­o­log­ic dys­func­tion that pre­vents nor­mal clot­ting in pa­tients’ blood.

Roche al­so has at least one big ad­van­tage in its fa­vor: Their drug—an an­ti-fac­tor IXa/X bis­pe­cif­ic which mim­ics the func­tion of Fac­tor VI­II, even in the pres­ence of FVI­II in­hibitors—is al­ready well in­to a ma­jor, Roche-style Phase III pro­gram with a drug blessed by the FDA with a break­through drug des­ig­na­tion. Two Phase III stud­ies have be­gun in chil­dren and adults who have ac­quired Fac­tor VI­II in­hibitors. An­oth­er Phase III in pa­tients with­out the FVI­II in­hibitors is start­ing, and there’s a fourth Phase III planned for a once-every-4-weeks dose, to see if they can come up with an eas­i­er ad­min­is­tra­tion sched­ule than the once-week­ly in­jec­tion they’re work­ing with now.

In­ves­ti­ga­tors say that their fol­lowup of 18 pa­tients in the key proof of con­cept study re­vealed that emi­cizum­ab cut the bleed­ing rate of pa­tients by more than 95% af­ter a fol­lowup re­view last­ing up to 32.6 months long. And that’s what they ex­pect to prove in Phase III.

He­mo­phil­ia A pa­tients lack the Fac­tor VI­II pro­tein re­quired for clot­ting, mak­ing a ge­net­ic fix one of the first tar­gets – along with he­mo­phil­ia B – se­lect­ed by a wave of gene ther­a­py com­pa­nies jump­ing in­to the game. But while Bio­Marin has seized the lead with its he­mo­phil­ia A gene ther­a­py, it still has a long way to go be­fore prov­ing that it can be done safe­ly over a pro­longed pe­ri­od of time.

Still, its treat­ment demon­strat­ed a star­tling abil­i­ty to boost Fac­tor VI­II in pa­tients, with in­ves­ti­ga­tors track­ing Fac­tor VI­II lev­els in sev­er­al pa­tients break­ing the 100% mark. Bleed­ing episodes were dra­mat­i­cal­ly re­duced, but not elim­i­nat­ed.

So the next time you hand­i­cap the race on he­mo­phil­ia drugs, where Bio­gen, Shire and oth­ers are scram­bling to pro­tect large fran­chis­es, don’t for­get Roche, which has the most ad­vanced drug in the clin­ic.

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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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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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.

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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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.

Mi­rati's KRAS drug looks like the ear­ly fa­vorite in colon can­cer with new da­ta, putting the pres­sure square on Am­gen

With Amgen already providing proof-of-concept for KRAS inhibitors with its sotorasib, Mirati Therapeutics is piecing together a follow-up effort in lung cancer with data it thinks are superior. But in colon cancer, where solo sotorasib has turned in a dud, Mirati may now have a strong case for superiority.

Mirati’s adagrasib, dosed solo or in combination with chemotherapy cetuximab, showed numerically higher response rates than sotorasib solo and as part of a combination study in a similar patient population also revealed this week at #ESMO21. Mirati’s data were presented as part of a cohort update from the Phase II KRYSTAL-1 study testing adagrasib in a range of solid tumors harboring the KRAS-G12C mutation.

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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.