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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

Endpoints Premium

Premium subscription required

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

Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Patty Murray, D-WA (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,200+ biopharma pros reading Endpoints daily — and it's free.

FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.

The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.

Peter Marks (Greg Nash/Pool via AP)

Even FDA's Pe­ter Marks is wor­ried about the com­mer­cial vi­a­bil­i­ty of gene and cell ther­a­pies

When bluebird bio’s gene therapy to treat beta thalassemia won European approval in 2019, the nearly $2 million per patient price tag for the potential cure seemed like a surmountable hurdle.

Fast forward two years later, and bluebird has withdrawn Zynteglo, the beta thal drug, along with the rest of its gene therapy portfolio from Europe, which the company said is generally unwilling to pay a fair price for the treatment.

Pri­cy in­halers re­main ex­pen­sive due to de­vice tweaks that keep com­peti­tors at bay, re­searchers find

New research published in Health Affairs today highlights the way in which the FDA’s inhaler regulations have rewarded incremental adjustments to older products, thereby enabling companies to skirt around cheaper competition.

A DC appeals court clerk and researchers from Harvard and the University of Calgary dug through all the patents and regulatory exclusivities granted to inhalers approved by the FDA between 1986 and 2020, finding that of the 62 inhalers approved, 53 (or 85%) were brand-name products, with a median of 16 years of protection from generic competition.

A CRL lat­er, Gilead clears clin­i­cal hold on next-gen HIV drug — af­ter find­ing a new glass vial for it

Back in late December, the FDA flagged concerns about the glass vials Gilead used to contain its experimental HIV therapy lenacapavir, citing risks of forming sub-visible glass particles in the drug solution.

Those issues were serious enough to trigger a clinical hold on 10 trials — and ultimately a rejection.

Two months after the complete response letter, Gilead said it’s finally found a solution that satisfies the FDA, which is lifting the clinical hold.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,200+ biopharma pros reading Endpoints daily — and it's free.

Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.