Alk­er­mes claims a suc­cess in PhI­II schiz­o­phre­nia head-to-head, but where’s the ben­e­fit for pa­tients?

Richard Pops

Alk­er­mes $ALKS has one shot at mak­ing ALKS-3831 a stand­out in the schiz­o­phre­nia field. For years now, in­ves­ti­ga­tors ex­pect­ed to be able to show that the drug per­forms pret­ty much the same as the gener­ic olan­za­p­ine in terms of ef­fi­ca­cy, but sig­nif­i­cant­ly bet­ter in terms of pre­vent­ing the weight gain — and all the risks that come with it — that af­flicts pa­tients on the old drug.

But in the first of two Phase III read­outs on Thurs­day evening, in­ves­ti­ga­tors were on­ly able to point to a sim­i­lar ef­fi­ca­cy with olan­za­p­ine with­out the ben­e­fit on weight gain.

El­liot Ehrich

Alk­er­mes CEO Richard Pops and R&D chief El­liot Ehrich were stick­ing with the top line and her­ald­ed the da­ta as a suc­cess. But in a wrap on side ef­fects the com­pa­ny al­so not­ed that weight gain was sim­i­lar for both drugs.

The stock dropped 5% as the da­ta hit Thurs­day evening, carv­ing out more than a bil­lion dol­lars in mar­ket cap.

“Those rates were sim­i­lar,” not­ed Ehrich dur­ing the Q&A with an­a­lysts Thurs­day evening.

The two ex­ecs, though, in­sist­ed that the 4-week study was sim­ply too short to ex­pect a sep­a­ra­tion on weight gain. That key ques­tion, they say, will have to wait for a 6-month study where pa­tients will have a chance to bal­ance the scale in fa­vor of the Alk­er­mes drug.

Based on its ear­li­er work, says Pops, it clear­ly takes time – three to six months — for the weight gain ad­van­tage to demon­strate it­self.

“Many physi­cians rec­og­nize the pow­er­ful ef­fi­ca­cy pro­file of olan­za­p­ine, but are hes­i­tant to pre­scribe it giv­en the se­vere weight gain and meta­bol­ic side ef­fects com­mon­ly as­so­ci­at­ed with its use,” said Christoph Cor­rell, M.D., pro­fes­sor of Psy­chi­a­try and Mol­e­c­u­lar Med­i­cine at Hof­s­tra North­well School of Med­i­cine. “A new an­tipsy­chot­ic with ro­bust ef­fi­ca­cy and a fa­vor­able weight and meta­bol­ic pro­file com­pared to olan­za­p­ine would be a wel­come ad­di­tion to the schiz­o­phre­nia treat­ment land­scape. This study con­firms a key el­e­ment of this pro­file, with a clear demon­stra­tion of ef­fi­ca­cy in a large, well-con­duct­ed clin­i­cal tri­al.”

No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Demis Hassabis, DeepMind CEO (Qianlong/Imaginechina via AP Images)

Google's Deep­Mind opens its pro­tein data­base to sci­ence — po­ten­tial­ly crack­ing drug R&D wide open

Nearly a year ago, Google’s AI outfit DeepMind announced they had cracked one of the oldest problems in biology: predicting a protein’s structure from its sequence alone. Now they’ve turned that software on nearly every human protein and hundreds of thousands of additional proteins from organisms important to medical research, such as fruit flies, mice and malaria parasite.

The new database of roughly 350,000 protein sequences and structures represents a potentially monumental achievement for the life sciences, one that could hasten new biological insights and the development of new drugs. DeepMind said it will be free and accessible to all researchers and companies.

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

No­var­tis dis­cards one of its ‘wild card’ drugs af­ter it flops in key study. But it takes one more for the hand

Always remember just how risky it is to gamble big on small studies.

A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy.

By last fall, the program had risen up to become one of CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won.

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6 top drug­mak­ers of­fer per­spec­tives on FDA's new co­vari­ates in RCTs guid­ance

Back in May, the FDA revised and expanded a 2019 draft guidance that spells out how to adjust for covariates in the statistical analysis of randomized controlled trials.

Building on the ICH’s E9 guideline on the statistical principles for clinical trials, the 3-page draft was transformed into an 8-page draft, with more detailed recommendations on linear and nonlinear models to analyze the efficacy endpoints in RCTs.

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In­side Bio­gen's scram­ble to sell Aduhelm: Pro­ject 'Javelin' and pres­sure to ID as many pa­tients as pos­si­ble

In anticipation of Aduhelm’s approval for Alzheimer’s in June, Biogen employees were directed to identify and guarantee treatment centers would administer the drug through a program called “Javelin,” a senior Biogen employee told Endpoints News.

The program identified about 800 centers for use, he said, and Biogen now pays for the use of bioassays to identify beta amyloid in potential patients having undergone a lumbar puncture procedure, the employee said — and one center preparing to administer the drug confirmed its participation in the bioassay program.

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EMA re­jects FDA-ap­proved Parkin­son's drug, signs off on Mod­er­na vac­cine use in ado­les­cents ahead of FDA

The European Medicines Agency on Friday rejected Kyowa Kirin’s Parkinson’s disease drug Nouryant (istradefylline), which the US FDA approved in 2019 under the brand name Nourianz.

EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”

Laurent Fischer, Adverum CEO

Ad­verum faces murky fu­ture af­ter re­view turns up deep­er safe­ty is­sues for gene ther­a­py

Three months after revealing that a patient lost significant vision in one eye after receiving its experimental gene therapy, Adverum announced it found the safety issues were more widespread: Five of 12 patients who received a high dose of the therapy saw “similar clinically-relevant events.”

Three required surgery on their treated eye. And all 12 are being recommended “aggressive immunomodulatory treatments” to prevent further injury.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

Bio­gen has a shaky end to H1 with a $542M write-off adding to its woes — but an­a­lysts see big rev­enue ahead for Aduhelm

All eyes at Biogen’s Q2 earnings call Thursday were on Aduhelm, but investors also got a glimpse of what Biogen would have faced had the FDA not opted to approve their controversial Alzheimer’s drug.

That glimpse, revealing a combination of declining sales, growing competition and failed medicines, underscores the stakes of the big biotech’s Aduhelm efforts, as execs punch back at the criticism they’ve engendered in the political and medical world and vigorously pushes its sales staff to roll out the drug as fast as possible.

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Mol­e­c­u­lar Di­ag­nos­tics Can Trans­form Can­cer Care. Let’s Make It Hap­pen.

Like so many people around the world, my life has been profoundly shaped by cancer. Those personal experiences, along with a deep love of clinical laboratory science and a passion to apply the power of genomics in medicine, motivated me to launch a company that would improve cancer care through better diagnostics. Thirteen years later, I am proud that we are delivering more accurate information at multiple points along the patient journey, with a focus on eight of the 10 cancers that are most commonly diagnosed in the United States.