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.”

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications.

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Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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Pur­due Phar­ma pleads guilty in fed­er­al Oxy­Con­tin probe, for­mal­ly rec­og­niz­ing it played a part in the opi­oid cri­sis

Purdue Pharma, the producer of the prescription painkiller OxyContin, admitted Tuesday that, yes, it did contribute to America’s opioid epidemic.

The drugmaker formally pleaded guilty to three criminal charges, the AP reported, including getting in the way of the DEA’s efforts to combat the crisis, failing to prevent the painkillers from ending up on the black market and encouraging doctors to write more painkiller prescriptions through two methods: paying them in a speakers program and directing a medical records company to send them certain patient information. Purdue’s plea deal calls for $8.3 billion in criminal fines and penalties, but the company is only liable for a fraction of that total — $225 million.

News brief­ing: Gilead part­ner Gala­pa­gos sells off CRO for $37M; Polyphor bags $3.3M from CF Foun­da­tion

Close Gilead ally Galapagos is selling off one of its contract research organizations to a Polish pharma company.

Galapagos has agreed to sell 100% of the outstanding shares in the CRO Fidelta to Selvita, in a deal worth roughly $37 million expected to close in the first week of January. The acquisition is expected to nearly double Selvita’s revenues, the company says, as well as expand its drug discovery efforts.