Or­chard Ther­a­peu­tics prunes jobs to re­fo­cus pri­or­i­ties on big­ger fruit

In 2018, Or­chard Ther­a­peu­tics se­cured ac­cess to GSK’s pi­o­neer­ing gene ther­a­py for “bub­ble boy syn­drome,” Strimvelis, among a bas­ket of oth­er rare dis­ease pro­grams, af­ter the big British drug­mak­er de­cid­ed the slate of drugs was too niche for its ar­se­nal of treat­ments. Now, Or­chard has al­so elect­ed to steer away from “ul­tra-rare” dis­eases to pro­grams fo­cused on more preva­lent con­di­tions.

The strate­gic shift, which al­so in­cludes halt­ing con­struc­tion of a Fre­mont man­u­fac­tur­ing fa­cil­i­ty and shut­ter­ing its Cal­i­for­nia R&D fa­cil­i­ty, has trig­gered a 25% cut in the com­pa­ny’s work­force.

“In our view, the new busi­ness plan rep­re­sents a smart repri­or­i­ti­za­tion of its com­mer­cial strat­e­gy to fo­cus on high­er yield op­por­tu­ni­ties and con­serve cash. This should re­move any need for near-term fi­nanc­ings,” Cowen’s Yaron Wer­ber wrote in a note.

Bob­by Gas­par

The Lon­don, Boston, and San Fran­cis­co-based biotech — which went pub­lic in 2018 in a bumper $200 mil­lion IPO — has a port­fo­lio of ex vi­vo gene ther­a­pies, in which a pa­tient’s stem cells are ex­tract­ed and tweaked out­side the body be­fore be­ing trans­plant­ed back in, with the aim to treat a va­ri­ety of rare im­mune and meta­bol­ic dis­eases. Founder and now chief Bob­by Gas­par was heav­i­ly in­volved in the de­vel­op­ment of these hematopoi­et­ic stem cell gene ther­a­pies, bring­ing them from some of the first stud­ies in pa­tients to po­ten­tial reg­u­la­to­ry ap­provals.

GSK took a near­ly 20% stake in Or­chard in 2018, in re­turn for Strimvelis and oth­er gene-ther­a­py pro­grams. Strimvelis was one of the few ap­proved gene ther­a­pies in 2016, but up­take in the rare pa­tient pop­u­la­tion for adeno­sine deam­i­nase se­vere com­bined im­mun­od­e­fi­cien­cy (ADA-SCID) had strug­gled — the ther­a­py had on­ly been used in a hand­ful of pa­tients by 2018. In its IPO prospec­tus, Or­chard had stressed that the ther­a­py’s prod­uct rev­enue would not be enough to make the com­pa­ny prof­itable. Be­sides, Or­chard was al­so de­vel­op­ing its own ACA-SCID gene ther­a­py, OTL-101.

On Thurs­day, Or­chard said it was re­duc­ing in­vest­ment in OTL-101 and OTL-300 (its trans­fu­sion-de­pen­dent be­ta-tha­lassemia pro­gram) — to pour its re­sources in­to its pro­grams for metachro­mat­ic leukody­s­tro­phy (OTL-200), Wiskott-Aldrich syn­drome (OTL-103), Mu­copolysac­cha­ri­do­sis type I (OTL-203) and Mu­copolysac­cha­ri­do­sis type II­IA, al­so known as San­fil­ip­po syn­drome type A (OTL-201). The com­pa­ny al­so un­veiled two new pre­clin­i­cal pro­grams in fron­totem­po­ral de­men­tia and Crohn’s dis­ease.

Or­chard’s US ap­pli­ca­tion to mar­ket OTL-200 may be de­layed, the com­pa­ny in­di­cat­ed on Thurs­day, as the FDA has raised ques­tions about the clin­i­cal end­points used, nat­ur­al his­to­ry da­ta and chem­istry man­u­fac­tur­ing and con­trol da­ta. The drug­mak­er plans to en­gage with the FDA this year to rem­e­dy these hic­cups be­fore mak­ing its sub­mis­sion, it said.

“As EMA raised sim­i­lar ques­tions about the clin­i­cal da­ta for OTL-200, man­age­ment is hope­ful that they have the da­ta that FDA is re­quest­ing. How­ev­er, as the meet­ing with the FDA is un­like­ly to oc­cur un­til some time in H2:20, we an­tic­i­pate that the BLA fil­ing will get de­layed,” Wer­ber wrote.

If a new phase I/II study is re­quired, the BLA fil­ing could be de­layed, he said, post­pon­ing his ex­pect­ed launch to 2024 from 2022.

Or­chard’s cash, cash equiv­a­lents and in­vest­ments as of March 31 were $263.9 mil­lion. The re­or­ga­ni­za­tion an­nounced on Thurs­day, which gen­er­at­ed cash sav­ings of ap­prox­i­mate­ly $125 mil­lion through the end of 2021, will ex­tend the com­pa­ny’s abil­i­ty to keep the lights on in­to 2022.

So­cial: Bob­by Gas­par, Or­chard CEO (Roy­al Col­lege of Pae­di­atrics and Child Health, YouTube)

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,700+ biopharma pros reading Endpoints daily — and it's free.

US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.

Almirall is tapping artificial intelligence on behalf of its sales force for insights and efficiencies. (via Shutterstock)

Almi­rall rolls out sales rep ar­ti­fi­cial in­tel­li­gence sys­tem, cut­ting pre-call prep and 'wind­shield time'

Dermatology specialty pharma Almirall is making its sales reps smarter. Not with extra training or educational courses, but instead with artificial intelligence tools.

It began a soft launch of a sales rep AI and machine learning platform it calls Polaris last August in one of its 7 US coverage regions. The platform from Aktana gathers information from across Almirall internal sources and external ones – such as claims and prescribing data – to generate insights for reps. Now, instead of spending hours prepping for a sales call, Polaris can generate details about a physician’s preferences, past behaviors and prescription habits for reps in minutes, said Almirall head of commercial operations Vincent Cerio.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,700+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er and CD­MOs ramp up Paxlovid man­u­fac­tur­ing with Kala­ma­zoo plant ex­pan­sion lead­ing the way

As the Covid-19 pandemic continues to evolve, pharma companies and manufacturers are exploring how to step up production on antivirals.

Pfizer is planning to expand its Kalamazoo-area facility to increase manufacturing capabilities for the oral Covid-19 antiviral Paxlovid, according to a report from Michigan-based news site MLive. The expansion of the facility, which serves as Pfizer’s largest manufacturing location, is expected to create hundreds of “high-skilled” STEM jobs, MLive reported. No details about the project’s cost and timeline have been released, but according to MLive, Pfizer will announce the details of the expansion at some point in early June.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,700+ biopharma pros reading Endpoints daily — and it's free.

FDA spells out the rules and re­stric­tions for states seek­ing to im­port drugs from Cana­da

The FDA is offering more of an explanation of the guardrails around its program that may soon allow states to import prescription drugs in some select circumstances from Canada, but only if such imports will result in significant cost reductions for consumers.

While the agency has yet to sign off on any of the 5 state plans in the works so far, and PhRMA’s suit to block the Trump-era rule allowing such imports is stalled, the new Q&A guidance spells out the various restrictions that states will have to abide by, potentially signaling that a state approval is coming.

Greg Mayes, Antios Therapeutics CEO

An­tios' HBV col­lab axed af­ter clin­i­cal hold, but biotech be­lieves safe­ty in­ci­dent is not treat­ment-re­lat­ed

The FDA has placed a clinical hold on a Phase IIa study of Antios Therapeutics’ investigational hepatitis B med, CEO Greg Mayes confirmed to Endpoints News in an emailed statement.

A safety report was delivered to the biotech on May 17 after a patient dosed in a triple combination cohort of the study had experienced bradycardia and hypotension. The triple combo included Antios’ ATI-2173, Assembly Biosciences’ vebicorvir and Viread, an approved antiviral for HIV and hepatitis B.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,700+ biopharma pros reading Endpoints daily — and it's free.

Tim Schoen, BioMed Realty CEO

Life sci­ences de­vel­op­er Bio­Med Re­al­ty buys San Fran­cis­co ho­tel for $75M — re­port

In a somewhat unconventional deal, life sciences real estate developer BioMed Realty has bought a 169-room Hilton Garden Inn in South San Francisco for $75 million, the San Francisco Business Times reported.

BioMed Realty, an affiliate of Blackstone, has multiple life sciences and technology office projects in the Bay Area, including three sites within a five-minute drive of the hotel.

While the sale of the hotel property was announced earlier this month, the sellers, Summit and GIC, did not identify the buyer at the time.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,700+ biopharma pros reading Endpoints daily — and it's free.

Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,700+ biopharma pros reading Endpoints daily — and it's free.