So­bi's high-pri­or­i­ty rare dis­ease drug runs in­to a wall at EMA

The EMA has re­buffed Swe­den-based So­bi’s pitch for ema­palum­ab, shoot­ing down high hopes for a $568 mil­lion drug.

So­bi said it will re­quest a re-ex­am­i­na­tion by the reg­u­la­tors, which will kick off a new re­view process that should pro­duce a de­ci­sion by the end of the year. The an­ti­body is de­signed to treat pri­ma­ry he­mo­phago­cyt­ic lym­pho­his­ti­o­cy­to­sis in chil­dren un­der 18 years old by tar­get­ing in­ter­fer­on gam­ma (IFNγ).

The drug, al­ready ap­proved in the US as Gam­i­fant, is one of the stars of So­bi’s pipeline af­ter it re­struc­tured its op­er­a­tions to fo­cus on hema­tol­ogy and im­munol­o­gy.

The com­pa­ny first li­censed it from Swiss biotech Novim­mune in 2018, pay­ing $50 mil­lion and promis­ing $400 mil­lion in mile­stones be­fore de­cid­ing to ac­quire the whole thing — com­plete with in­tel­lec­tu­al prop­er­ty rights, the team work­ing on it and a pri­or­i­ty re­view vouch­er. Un­der the new deal, mile­stones add up to $518 mil­lion.

“Dur­ing the last years our team has gained a lot of ex­pe­ri­ence in this rather com­plex dis­ease area,” CEO Gui­do Oelk­ers said in a state­ment, as over 100 pa­tients have been treat­ed in the US.

He didn’t elab­o­rate on what might have gone wrong in the ap­pli­ca­tion. Fran­co Lo­catel­li, the prin­ci­pal in­ves­ti­ga­tor in the EU, was quot­ed say­ing he was “sig­nif­i­cant­ly sur­prised” about the EMA rec­om­men­da­tion against ap­proval.

“I had the priv­i­lege to ob­serve that this mon­o­clon­al an­ti­body, tar­get­ing the main cy­tokine in­volved in the dis­ease patho­phys­i­ol­o­gy, was well tol­er­at­ed and ef­fec­tive in a large pro­por­tion of the pa­tients, rep­re­sent­ing a mod­el of pre­ci­sion med­i­cine,” he said. “While US chil­dren have since al­most 2 years the pos­si­bil­i­ty to be treat­ed with this nov­el, safe, high­ly ef­fec­tive and tar­get­ed ther­a­py, the EMA de­ci­sion paves the way for mi­gra­to­ry health flows to­wards non-Eu­ro­pean Cen­ters that can grant this treat­ment.”

In the re­lease So­bi al­so re­it­er­at­ed its es­ti­mat­ed peak sales tar­get of over $500 mil­lion — which “re­mains un­changed re­gard­less of an ap­proval in Eu­rope.”

Sales of Gam­i­fant in the first six months of 2020 were $26.7 mil­lion. In 2019, the first year of launch, to­tal sales were $61.3 mil­lion.

By So­bi’s es­ti­mate, the most im­por­tant mar­kets based on num­ber of pa­tients for HLH — a rare but se­vere dis­ease that can re­sult in fever, en­large­ment of the liv­er and spleen, en­larged lymph nodes, rash­es and hos­pi­tal­iza­tions — are Chi­na, fol­lowed by the US, Eu­rope and Japan.

It’s al­so ini­ti­at­ing clin­i­cal stud­ies for pre-emp­tive treat­ment of pa­tients with risk fac­tors of HSCT acute graft fail­ure.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

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What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Kimberly Smith, ViiV R&D chief (ViiV Healthcare)

Af­ter sting­ing FDA set­back, Glax­o­SmithK­line's Vi­iV fi­nal­ly notch­es US ap­proval for long-act­ing HIV in­jec­tion

GlaxoSmithKline’s HIV unit ViiV was dealt a stinging loss back in late 2019 when the FDA slammed the brakes on its application for a once-monthly injection based on manufacturing issues. Now, with that roadblock in the rearview, ViiV has finally made good on its promise to change the HIV game.

The FDA on Thursday approved ViiV’s Cabenuva (cabotegravir and rilpivirine) as a long acting, once-monthly therapy for HIV-positive adults who are virologically suppressed and on a stable antiviral regimen.

Michelle McMurry-Heath, BIO CEO (BIO via YouTube)

BIO looks to re­struc­ture, lay­ing off staff amid chal­lenge to the trade org's nor­mal face-to-face style

The biopharma industry, on the whole, had a red-letter year in 2020 amid Covid-19, with fundraising at an all-time high and major players speeding vaccines ahead to approval. But for BIO, the industry’s leading trade organization, the pandemic has prompted a reconsideration of the game plan.

BIO will pivot to digital as the Covid-19 pandemic continues to rage, making “some staff reductions” as it looks to bring its roughly 37,000 in-person meetings each year to the web, the organization said Thursday.

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Fast on Glax­o­SmithK­line's heels, Au­rinia wins OK to steer a sec­ond lu­pus nephri­tis drug straight to the mar­ket

GlaxoSmithKline’s Benlysta isn’t alone in the small circle of approved lupus nephritis drugs anymore.

Little Aurinia Pharmaceuticals has gotten the green light from the FDA to start marketing its first and only program, voclosporin, under the brand name Lupkynis — something CEO Peter Greenleaf says it’s been ready to do since December.

Regulators went right down to the wire on the decision, keeping the company and the entire salesforce it’s already assembled on its toes.

Cardiovascular chief Joerg Koglin (Merck)

Mer­ck makes good on big CV gam­ble with veri­ciguat nod — but com­pe­ti­tion will be fierce

The results for Merck’s leading heart drug fell short of what some cardiologists had hoped for, but they proved more than enough to convince the FDA, which handed down an approval Wednesday.

The agency OK’d vericiguat, now marketed as Verquvo, for patients with heart failure, offering one of the first new pharmaceutical tools to tackle a hard-to-treat group of patients at high risk for what remains the leading cause of death in the US and Europe. It’s also a notable win for Merck, who rolled the dice on the molecule in 2016, paying Bayer $1 billion for US rights to the then-experimental drug and ex-US rights for the approved CV drug Adempas.

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