FDA hits Su­per­nus with dou­ble wham­my, hand­ing biotech a CRL and RTF on same day

Su­per­nus had a bad Mon­day.

Yes­ter­day, the Mary­land biotech an­nounced that two dif­fer­ent ther­a­pies had been shot back by the FDA. The agency re­ject­ed their ap­pli­ca­tion to ap­prove SPN-812, an ex­per­i­men­tal AD­HD med­ica­tion for kids and ado­les­cents. And they re­fused to even con­sid­er an ap­pli­ca­tion for SPN-830, a con­tin­u­ous treat­ment pump meant for Parkin­son’s dis­ease.

The re­jec­tion is a sig­nif­i­cant blow to Su­per­nus, which in­vest­ed in four clin­i­cal tri­als for the AD­HD med. The FDA doesn’t dis­close its rea­son­ing for re­ject­ing a drug, but the com­pa­ny said it had to do with CMC is­sues, rather than safe­ty or ef­fi­ca­cy con­cerns. An in-house lab that does an­a­lyt­ic test­ing re­cent­ly changed lo­ca­tions, they said, rais­ing ques­tions in the agency about qual­i­ty con­trol.

Su­per­nus said they plan to “clar­i­fy to the FDA” that they do “not re­ly sole­ly on this fa­cil­i­ty” for qual­i­ty con­trol and then dis­cuss how to re­sub­mit their ap­pli­ca­tion.

The com­pa­ny was less forth­com­ing on why the agency re­fused to file their Parkin­son’s ap­pli­ca­tion. “In the let­ter, the FDA re­quest­ed cer­tain doc­u­ments and re­ports to be sub­mit­ted in sup­port of the ap­pli­ca­tion,” the com­pa­ny said.

Su­per­nus stock $SUPN fell 18%  on the news pre-mar­ket, from 24.95 to 20.35.

Jack Khat­tar

Ex­pec­ta­tions around the AD­HD drug, how­ev­er, had al­ready be­gun to wane. Su­per­nus CEO Jack Khat­tar has billed SPN-812 as the “first tru­ly new ther­a­py” for AD­HD in a decade, but the re­sults in late-stage tri­als fell short of ex­pec­ta­tions.

Al­though pos­i­tive, their first cut of the Phase III study ap­peared to show an ef­fect size no greater than a ri­val Eli Lil­ly drug and failed to show a dose-de­pen­dent re­sponse in the high dose, wor­ry­ing in­vestors. The com­pa­ny has point­ed to da­ta sug­gest­ing their treat­ment can work faster, with pa­tients feel­ing the ef­fect af­ter one week as op­posed to sev­er­al weeks.

The SPN-830 Parkin­son’s pump was part of their $300 mil­lion buy­out of the Ken­tucky-based neu­ro­science com­pa­ny US WorldMeds. Su­per­nus en­vi­sioned it as an al­ter­na­tive to more in­va­sive pro­ce­dures for pa­tients strug­gling with symp­toms, such as the gas­tric tubes some­times used to pro­vide con­tin­u­ous dos­ing of L-Dopa.

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

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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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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.”

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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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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Severin Schwan (AP Images)

Roche CEO Schwan puts 3-time Alzheimer's los­er gan­tenerum­ab back in the spot­light as FDA's con­tro­ver­sial Aduhelm OK in­spires block­buster fore­casts

Four years after Roche opted to resurrect its late-stage effort on their Alzheimer’s program for gantenerumab following a clear failure, Roche CEO Severin Schwan is signaling some fresh enthusiasm for its blockbuster prospects in the wake of the controversial Aduhelm OK.

Schwan told reporters that company execs are engaged in ongoing talks with the FDA as speculation continues to percolate around its chances based on biomarker data alone, now that regulators have established a precedent using an accelerated approval pathway process with no convincing evidence of efficacy in helping patients retain their cognitive skills. In Aduhelm’s case, the drug reduced the amount of amyloid in the brain and Biogen was able to win the accelerated approval on what the FDA called a reasonable expectation of success.

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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.

Mod­er­na es­tab­lish­es pub­lic health-fo­cused char­i­ty; FDA ap­proves As­traZeneca di­a­betes drug for pe­di­atric use

To help promote public health and healthcare in underserved areas of the world, Moderna will establish a charity with a $50 million endowment.

The Cambridge, MA-based company announced the board of directors’ approval Thursday. The foundation will focus on “charitable, scientific and educational endeavors” with an emphasis on promoting public health and the access to healthcare, the press release said. The foundation will start operations once its status as a 501(c)(3) is approved.