Shire’s multi­bil­lion-dol­lar gam­ble on lanadelum­ab pays off with stel­lar PhI­II da­ta, quick mar­ket­ing ap­pli­ca­tion

Flem­ming Orn­skov

When Shire bought out Dyax for up to $6.5 bil­lion in 2015, the big prize was a new drug for hered­i­tary an­gioede­ma dubbed DX-2930 which posed a di­rect threat to Cin­ryze, a fran­chise drug for the rare dis­ease play­er.

Thurs­day morn­ing, Shire CEO Flem­ming Orn­skov says their mul­ti-bil­lion dol­lar bet paid off with stel­lar Phase III da­ta for the drug, now called lanadelum­ab (SHP643).

A 300 mg dose of the drug twice a month de­liv­ered an 87% re­duc­tion in mean HAE at­tack fre­quen­cy, com­pared to 0% in the place­bo group. It hit the pri­ma­ry end­point with “high­ly” sta­tis­ti­cal­ly sig­nif­i­cant re­sults as well as all the sec­on­daries.

Al­ready des­ig­nat­ed a break­through ther­a­py with or­phan drug sta­tus, Shire says it will now race to the FDA lat­er this year or ear­ly next with a bi­o­log­ics ap­pli­ca­tion, with plans to cap­i­tal­ize on the re­sults as soon as pos­si­ble.

Shares of the $55 bil­lion biotech $SH­PG surged 6% on the news in pre-mar­ket trad­ing.

Many an­a­lysts be­lieve that Shire, which swelled in size af­ter it com­plet­ed the Bax­al­ta and Dyax ac­qui­si­tions, is on a short path to block­buster rev­enue. Shire it­self set a goal of $2 bil­lion in peak sales for this drug. And Eval­u­atePhar­ma re­cent­ly picked it as a top-10 or­phan drug bound for a boom­ing mar­ket with 6-fig­ure price tags.

“The pos­si­bil­i­ty of a new way to ad­dress the un­der­ly­ing cause of HAE to pre­vent at­tacks could trans­form how we treat the dis­ease in the fu­ture,” said Pro­fes­sor Mar­cus Mau­r­er, M.D., Char­ité –Uni­ver­sitätsmedi­zin Berlin, Ger­many and clin­i­cal tri­al in­ves­ti­ga­tor. “Pa­tients with HAE want to live in­de­pen­dent­ly and with­out fear of an an­gioede­ma at­tack.”

Shire paid close to $6 bil­lion for Dyax, large­ly on the promise of the next-gen HAE ther­a­py. And Orn­skov added a $4 CVR worth $656 mil­lion if the drug is ap­proved. That pay­off could be loom­ing in 2018 if the da­ta pass­es muster with reg­u­la­tors.

The Phase III re­sults mark an­oth­er big win for CEO Flem­ming Orn­skov, who was de­ter­mined to trans­form the com­pa­ny, based in Lex­ing­ton, MA., af­ter he took over. Shire went on to ex­pe­ri­ence some bumps along the road, but in the longterm his strate­gic plan and will­ing­ness to bet big on new drugs has been pay­ing off.

“We are ex­treme­ly en­cour­aged by these topline Phase III re­sults,” said Orn­skov in a state­ment, “We have near­ly a decade of ex­pe­ri­ence and a strong port­fo­lio and pipeline in HAE and be­lieve these da­ta demon­strate high po­ten­tial for trans­form­ing the way pa­tients liv­ing with this con­di­tion are treat­ed.”

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Reshma Kewalramani, Vertex CEO (Vertex via YouTube)

Bat­tling a line­up of skep­tics, Ver­tex claims an­oth­er ear­ly clin­i­cal win — this time in kid­ney dis­ease

Vertex claimed its second early-stage win of the fall Wednesday, announcing positive results in a small study on a genetically defined form of kidney disease.

The 16-patient, Phase II trial focused on patients with focal segmental glomerulosclerosis, a rare disease where kidneys are unable to filter blood properly. Over 13 weeks on an experimental pill, the level of protein in the patients’ urine fell by an average of 47.6%.

With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

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How to use reg­istry da­ta to sup­port FDA de­ci­sion mak­ing: Agency ex­plains in new guid­ance

Drugmakers looking to design a new registry or use an existing one to support a regulatory decision on a drug’s effectiveness or safety will need to consult with a new draft guidance released Monday by the FDA.

The agency’s reliance on registry data for regulatory decisions dates back more than two decades, at least, as in 1998 Bayer won approval for its anticoagulant Refludan (withdrawn from the market in 2013 for commercial reasons) based in part on a historical control group pulled from a registry.