Roche says its new in­ter­im up­date on he­mo­phil­ia A drug is pos­i­tive. But it’s hard to gauge.

Roche to­day sent out a small, blur­ry snap­shot of the in­ter­im da­ta from a Phase III study of emi­cizum­ab in chil­dren suf­fer­ing from he­mo­phil­ia A.

What did we find out?

San­dra Horn­ing

The da­ta are pos­i­tive and “clin­i­cal­ly mean­ing­ful” — though pre­sum­ably not sta­tis­ti­cal­ly sig­nif­i­cant, which is the phrase you look for in de­ter­min­ing suc­cess or fail­ure — in cut­ting the bleed rate among chil­dren 12 or younger with in­hibitors to Fac­tor VI­II.

The most com­mon ad­verse events: in­jec­tion site re­ac­tions and na­sopharyn­gi­tis.

Any se­ri­ous ad­verse events, like the ones seen in HAVEN 1?

In a fol­lowup, a spokesper­son tells me: “No throm­boem­bol­ic (TE) events or cas­es of throm­bot­ic mi­croan­giopa­thy (TMA) oc­curred in this study pop­u­la­tion (HAVEN 2).”

And as for the “clin­i­cal­ly mean­ing­ful” bot­tom line, she adds: “(G)iv­en the study de­sign is a sin­gle arm (no com­para­tor), we can’t say “sta­tis­ti­cal­ly sig­nif­i­cant” be­cause there is no hy­poth­e­sis test­ing, but the re­duc­tion is clin­i­cal­ly mean­ing­ful to treaters and pa­tients. This type of study de­sign is stan­dard for pe­di­atric stud­ies in he­mo­phil­ia. We look for­ward to shar­ing de­tails of the bleed rates when the da­ta are pre­sent­ed at con­gress. We’ve sub­mit­ted to ISTH in Ju­ly.”

Fair enough.

Roche’s re­cent suc­cess in gain­ing an ap­proval for Ocre­vus in mul­ti­ple scle­ro­sis set them on a di­rect course to dis­rupt­ing the mul­ti­ple scle­ro­sis field, with an­oth­er like­ly block­buster to add to a list that al­so in­cludes the PD-L1 check­point Tecen­triq. Emi­cizum­ab, bet­ter known as ACE910, is in­tend­ed to be the next on the list with peak sales es­ti­mates from an­a­lysts hov­er­ing around $1.4 bil­lion.

Two months ago Roche trig­gered fresh doubts about its drug af­ter a pa­tient in HAVEN 1 died fol­low­ing two se­ri­ous ad­verse events. The death spurred fresh ques­tions about the drug’s safe­ty when in­ves­ti­ga­tors had to fend off per­sis­tent ques­tions about 4 spon­ta­neous­ly re­port­ed SAEs af­ter two pa­tients had throm­boem­bol­ic events and two pa­tients de­vel­oped throm­bot­ic mi­croan­giopa­thy, or TMA. That news helped briefly buoy Shire and No­vo Nordisk, which both see a big ri­val to their block­buster he­mo­phil­ia fran­chis­es in emi­cizum­ab.

Two more Phase III stud­ies in the pro­gram are on­go­ing.

“Man­ag­ing haemophil­ia A with in­hibitors to fac­tor VI­II is es­pe­cial­ly chal­leng­ing for chil­dren and their care­givers, be­cause bleed­ing is dif­fi­cult to con­trol and cur­rent treat­ments re­quire fre­quent in­tra­venous in­fu­sions”, said San­dra Horn­ing, Roche’s CMO and prod­uct de­vel­op­ment chief. “We are en­cour­aged that once-week­ly sub­cu­ta­neous emi­cizum­ab pro­phy­lax­is showed a clin­i­cal­ly mean­ing­ful re­duc­tion in the num­ber of bleeds over time in chil­dren and are pleased to share these re­sults with the com­mu­ni­ty as we join in cel­e­brat­ing World He­mo­phil­ia Day.”

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

UP­DAT­ED: In a land­mark first glimpse of hu­man da­ta from Ver­tex, CRISPR/Cas9 gene ther­a­py sig­nals ear­ly ben­e­fit

Preliminary data on two patients with blood disorders that have been administered with Vertex and partner CRISPR Therapeutics’ gene-editing therapy suggest the technology is safe and effective, marking the first instance of the benefit of the use of CRISPR/Cas9 technology in humans suffering from disease.

Patients in these phase I/II studies give up peripheral blood from which hematopoietic stem and progenitor cells are isolated. The cells are tinkered with using CRISPR/Cas9 technology, and the edited cells — CTX001 — are infused back into the patient via a stem cell transplant. The objective of CTX001 is to fix the errant hemoglobin gene in patents with two blood disorders: beta-thalassemia and sickle cell disease, by unleashing the production of fetal hemoglobin.

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UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

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David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

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Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Stephen Hahn (via Senate HELP Committee)

Stephen Hahn gets through Sen­ate’s soft­ball job in­ter­view — but most­ly plays dodge­ball on the is­sues fac­ing the FDA

Anyone looking for fresh insights on what kind of FDA commissioner Stephen Hahn will be got precious few clues during Wednesday’s Senate hearing on the nomination.

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Op­di­vo/Yer­voy com­bo for melanoma fails in key pa­tient pop­u­la­tion

Bristol-Myers Squibb’s efforts to expand their checkpoint inhibitor combination have run into another recalcitrant cancer.

The NJ-based pharma announced that a combination of Yervoy and Opdivo didn’t beat out Opdivo alone in patients with resected high-risk melanoma who had very low levels of PD-L1. The drug combo couldn’t improve recurrence-free survival in these post-surgery patients.

Ver­tex's stel­lar quar­ter car­ries on with French re­im­burse­ment deal

Vertex’s golden quarter just got brighter. About a month after the US drugmaker finally clinched a deal with UK authorities to cover its slate of cystic fibrosis (CF) drugs following years of protracted negotiations, the company on Wednesday secured a deal with France for its CF therapy, Orkambi.

After the UK, France has one of the largest CF populations outside the United States. Achieving French reimbursement unlocks an ~7000-patient CF population, around ~2500-3000 of which will likely be eligible to receive (and be reimbursed for) Orkambi, Stifel’s Paul Matteis wrote in a note.

Nello Mainolfi, Kymera via Youtube

Kymera hands the helm to No­var­tis vet — and found­ing CSO — Nel­lo Main­olfi

Kymera Therapeutics is turning to a co-founder to run the company.
The protein degradation specialist with a deep-pocket syndicate behind them has opted to give the helm officially to Nello Mainolfi. The new CEO is a veteran of the Novartis Institutes for Biomedical Research. He joined Atlas Venture in their entrepreneur-in-residence program and helped launch Kymera as the CSO three years ago with Atlas’ Bruce Booth.
The boast at Kymera is that they’re angling to create a new class of protein degraders, a popular field where there’s been a variety of startups. One of its chief advocates is NIBR head Jay Bradner, who launched C4 just ahead of joining Novartis, where he’s also been doing new work in the field.