News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

“(T)here is cur­rent­ly no ap­proved ther­a­py for pa­tients who progress on CAR-Ts,” not­ed Re­gen­eron’s Is­rael Lowy. “Our po­ten­tial­ly reg­is­tra­tional Phase 2 pro­gram is ini­ti­at­ing this month and will proac­tive­ly eval­u­ate ac­tive REGN1979 dos­es in in­do­lent and ag­gres­sive non-Hodgkin lym­phoma.”

Not­ed Baird’s Bri­an Sko­r­ney:

The fol­lic­u­lar lym­phoma da­ta don’t look as stel­lar as the pri­or da­ta cut but the DL­B­CL da­ta im­prove. We think this bis­pe­cif­ic pro­gram looks like a strong next-gen B-cell lym­phoma ther­a­py that could yield a fair­ly sig­nif­i­cant mar­ket op­por­tu­ni­ty.

Ar­Qule rights the ship with more proof-of-con­cept da­ta

Af­ter a less than flat­ter­ing show­ing at the last EHA, Ar­Qule is re­turn­ing tri­umphant.

Hom­ing in on the BTK-C481AS mu­ta­tion, Ar­Qule high­light­ed da­ta from a co­hort of six chron­ic lym­pho­cyt­ic leukemia pa­tients who have ei­ther not re­spond­ed to treat­ment or re­lapsed. Hav­ing re­ceived the 65 mg dose, four of them ex­pe­ri­enced a par­tial re­sponse — a sig­nif­i­cant feat giv­en they have al­ready ex­haust­ed avail­able ther­a­pies, the com­pa­ny said. Shares $AR­QL surged 47% pre-mar­ket to $9.26.

Bri­an Schwartz Ar­Qule

“We are now fo­cused on fi­nal­iz­ing the rec­om­mend­ed phase 2 dose and plan­ning for the ex­pan­sion of our clin­i­cal ef­forts with ARQ 531 in­to lat­er stage tri­als across mul­ti­ple in­di­ca­tions as a sin­gle agent and as a com­bi­na­tion ther­a­py,” CMO Bri­an Schwartz said in a pre­pared state­ment.

In ad­di­tion, in­ves­ti­ga­tors al­so not­ed a par­tial re­sponse for a pa­tient with Richter’s Trans­for­ma­tion who had pro­gressed on ibru­ti­nib and R-CHOP.

Lit­tle Ory­zon shows it’s do­ing al­right with AML drug on its own

Ory­zon has seen the first glimpse of ef­fi­ca­cy da­ta for its LSD1 in­hibitor, iadadem­stat when com­bined with chemother­a­py in el­der­ly pa­tients with acute myeloid leukemia. Clin­i­cal re­spons­es were ob­served rapid­ly with a me­di­an time of 1.5 months, and of the 5 evalu­able pa­tients, 3 achieved com­plete re­mis­sions (al­beit with in­com­plete hema­to­log­ic re­cov­ery) while 1 had a par­tial re­mis­sion.

The tri­al is be­ing con­duct­ed in Spain, where Ory­zon was found­ed. The com­pa­ny plans to re­cruit up to 36 pa­tients to set the stage for the broad­er ap­pli­ca­tion of iadadem­stat —  once part­nered but lat­er dumped by Roche — in oth­er leukemias.

BerGen­Bio spot­lights AML drug’s po­ten­tial in pa­tients un­fit for in­tense chemo

Nor­way’s BerGen­Bio is spot­light­ing 6 re­spons­es out of 14 evalu­able pa­tients on its drug, which is de­signed to pair with low-in­ten­si­ty chemother­a­py for acute myeloid leukemia pa­tients who can­not stand in­tense chemother­a­py. Their AXL in­hibitor be­m­cen­tinib helped push 4 pa­tients in­to com­plete re­mis­sion (with or with­out in­com­plete hema­to­log­ic re­cov­ery) and 2 oth­ers in­to par­tial re­mis­sion.

“Be­m­cen­tinib in com­bi­na­tion with LDAC re­sult­ed in a sub­stan­tial­ly high­er ORR than ex­pect­ed for sin­gle-agent cy­tara­bine and clear­ly war­rant fur­ther in­ves­ti­ga­tion of be­m­cen­tinib in an ex­pan­sion co­hort of AML pa­tients un­fit for in­ten­sive chemother­a­py,” pro­fes­sor Bjørn Tore Gjert­sen of Hauke­land Uni­ver­si­ty Hos­pi­tal and the Uni­ver­si­ty of Bergen said. “Our sig­nal cell-lev­el bio­mark­er analy­sis of AML blasts from pa­tients in­di­cates a sub­stan­tial ef­fect on cell sig­nal­ing and rep­re­sents a po­ten­tial new bio­mark­er strat­e­gy.”

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Joe Jimenez, Getty

Ex-No­var­tis CEO Joe Jimenez is tak­ing an­oth­er crack at open­ing a new chap­ter in his ca­reer — and that in­cludes a new board seat and a $250M start­up

Joe Jimenez is back.

The ex-CEO of Novartis has taken a board seat on Century Therapeutics, the Versant and Bayer-backed startup focused on coming up with a brand new twist on cell therapies for cancer — a field where Jimenez made his mark backing the first personalized CAR-T approved for use.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,800+ biopharma pros reading Endpoints daily — and it's free.

Credit: Shutterstock

Can we make the an­tibi­ot­ic mar­ket great again?

The standard for-profit model in drug development is straightforward. Spend millions, even billions, to develop a medicine from scratch. The return on investment (and ideally a tidy profit) comes via volume and/or price, depending on the disease. But the string of big pharma exits and slew of biotech bankruptcies indicate that the model is sorely flawed when it comes to antibiotics.

The industry players contributing to the arsenal of antimicrobials are fast dwindling, and the pipeline for new antibiotics is embarrassingly sparse, the WHO has warned. Drugmakers are enticed by greener pastures, compared to the long, arduous and expensive path to antibiotic approval that offers little financial gain as treatments are typically priced cheaply, and often lose potency over time as microbes grow resistant to them.

Top Har­vard chemist caught up in FBI’s 'T­hou­sand Tal­ents' drag­net, ac­cused of ly­ing about Chi­nese con­nec­tions, pay

The FBI’s probe into the alleged theft of R&D secrets by Chinese authorities has drawn Harvard’s top chemist into its net.

The agency accused Charles M. Lieber, who chairs the university’s chemistry and chemical biology department, with lying about his involvement in China’s Thousand Talents campaign, which was established as a way of drawing in innovators from around the world. And the scientist, 60, was charged with making false statements about his ties to China.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,800+ biopharma pros reading Endpoints daily — and it's free.

Eye­ing a trio of tri­al ini­ti­a­tions, Jim Wilson's gene ther­a­py start­up woos Bruce Gold­smith from Deer­field as CEO

Passage Bio — Jim Wilson’s self-described “legacy company” — has wooed a seasoned biotech executive to steer the clinical entry of its first three gene therapy programs.

Bruce Goldsmith jumps to the helm of Passage after a brief CEO stint at Civetta, a cancer-focused startup he helped launch while a venture partner at Deerfield. He takes over from OrbiMed partner and interim chief Stephen Squinto, who will now lead the R&D team.

Amber Saltzman (Ohana)

Flag­ship's first ven­ture of 2020 is out, and it's all about sperm

A couple years ago, Amber Salzman got a call as she was returning East full-time after a two-year stint running a gene therapy company in California.

It was from someone at Flagship Pioneering, the deep-pocketed biotech venture firm. They had a new company with a new way of thinking about sperm. It had been incubating for over a year, and now they wanted her to run it.

“It exactly fit,” Salzman told Endpoints News. “I just thought I had to do something.”

Pfiz­er ax­es 6 ear­ly to late-stage can­cer stud­ies from the pipeline — with one oth­er cut for sick­le cell dis­ease

Pfizer trimmed a group of 3 R&D programs using their PD-L1 Bavencio — partnered with Merck KGaA — in their latest pipeline cull.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,800+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: In­cyte scores much need­ed PhI­II suc­cess — and of course it’s de­liv­ered by rux­oli­tinib

Incyte’s efforts to breathe a second life into ruxolitinib — its JAK inhibitor sold in pill form as Jakafi — has been greeted with clear, if preliminary and unsurprising, Phase III success.

Topline data from the TRuE-AD2 cements ruxolitinib’s foundational importance for Incyte, and gives analysts hope that there might yet be room for growth in a pipeline that’s suffered multiple R&D setbacks.

The FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. They plan to squash it — and per­ma­nent­ly ex­ile him

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition. And their lawsuit is asking that Shkreli — with several years left on his prison sentence — be banned permanently from the pharma industry.