Su­per­nus touts win­ning da­ta in adult AD­HD as it looks to make its case with the FDA — again — for pe­di­atric use

Less than two months af­ter be­ing re­ject­ed by the FDA for pe­di­atric pa­tients, Su­per­nus Phar­ma­ceu­ti­cals’ AD­HD pro­gram has new da­ta in an old­er pop­u­la­tion that the com­pa­ny hopes will re­verse its for­tunes. Will the agency bite?

Su­per­nus’ $SUPN SPN-812 im­proved adult pa­tients’ re­sults on an im­por­tant AD­HD symp­toms test at a sta­tis­ti­cal­ly sig­nif­i­cant rate high­er than place­bo, ac­cord­ing to topline Phase III re­sults post­ed Tues­day evening. The biotech had sought ap­proval in chil­dren ages 6 to 17, but was shot down in No­vem­ber af­ter reg­u­la­tors cit­ed con­cerns about an in-house lab used to an­a­lyze the ex­per­i­men­tal drug.

The news brought some ear­ly Christ­mas joy to in­vestors, as shares jumped rough­ly 16% in ear­ly Wednes­day trad­ing.

Su­per­nus is plot­ting to sub­mit the adult da­ta to the FDA as a pos­si­ble sup­ple­ment to its sought-af­ter ap­proval in pe­di­atric pa­tients — but that would re­quire a pos­i­tive re­sult from the phar­ma’s meet­ing with the agency in Jan­u­ary to dis­cuss that CRL from last month. If a turn­around ap­proval does come ear­ly next year, Su­per­nus said it would like­ly pe­ti­tion for an adult OK in the sec­ond half of 2021.

Re­searchers test­ed SPN-812 in 374 adults in a ran­dom­ized, dou­ble-blind­ed and place­bo-con­trolled set­ting. Pa­tients in the drug arm took the once-dai­ly pill for six weeks, start­ing with a 200-mg dose but with the op­tion to in­crease dosage up to 600 mg. To mea­sure ef­fi­ca­cy, Su­per­nus looked at the change from base­line to the end of the study on the AD­HD In­ves­ti­ga­tor Symp­tom Rat­ing Scale, or AISRS.

At the end of six weeks, those in the SPN-812 group saw a change of -15.5 in AISRS scores com­pared to -11.7 in the con­trol arm. Those fig­ures turned out a p-val­ue of p=0.0040, and Su­per­nus said the sta­tis­ti­cal sig­nif­i­cance was con­firmed lat­er by a sen­si­tiv­i­ty analy­sis show­ing a p-val­ue of p=0.0085.

The com­pa­ny al­so put forth da­ta it said showed the pro­gram in­di­cat­ed a fast on­set of ac­tion, reach­ing sta­tis­ti­cal sig­nif­i­cance af­ter just two weeks. Though Su­per­nus did not pro­vide change from base­line fig­ures, they said the p-val­ue fol­low­ing this pe­ri­od was p=0.0397.

Su­per­nus didn’t clar­i­fy how it planned to an­swer the FDA’s con­cerns out­lined in the No­vem­ber CRL to push for a pe­di­atric nod. The in-house lab that does an­a­lyt­ic test­ing had re­cent­ly changed lo­ca­tions pri­or to the agency’s re­view, Su­per­nus said, rais­ing ques­tions about qual­i­ty con­trol. Su­per­nus said at the time they aimed to clar­i­fy the lab is not the on­ly fa­cil­i­ty used to test the can­di­date.

No­vem­ber’s re­jec­tion was a sig­nif­i­cant blow for Su­per­nus, giv­en the com­pa­ny has con­duct­ed four tri­als in the AD­HD field. It al­so came hand in hand with the agency re­fus­ing to even con­sid­er an ap­pli­ca­tion for Su­per­nus’ SPN-830, a con­tin­u­ous treat­ment pump meant for Parkin­son’s dis­ease.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

Credit: Shutterstock

How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

Ugur Sahin, AP Images

As pres­sure to share tech­nol­o­gy mounts, BioN­Tech se­lects Rwan­da for lat­est vac­cine site

BioNTech’s first mRNA-based vaccine site in Africa will call Rwanda home, and construction is set to start in mid-2022, the company announced Tuesday at a public health forum.

The German company signed a memorandum of understanding, after a meeting between Rwanda’s Minister of Health, Daniel Ngamije, Senegal’s Minister of Foreign Affairs Aïssata Tall Sall, and senior BioNTech officials. Construction plans have been finalized, and assets have been ordered. The agreement will help bring end-to-end manufacturing to Africa, and as many as several hundred million doses of vaccines per year, though initial production will be more modest.

UP­DAT­ED: Eli Lil­ly toss­es a mar­quee pain drug and hits the gas on Alzheimer’s — as Bio­gen’s suf­fer­ing opens mar­ket to ri­vals

The furious chorus of critics that brought sales of Biogen’s ultra controversial Alzheimer’s drug aducanumab (sold as Aduhelm) to a near halt is opening up some big opportunities for a major league rival that has long sought the lead role in this largely untapped megamarket.

In its Q3 update today, Eli Lilly — noted for its dogged persistence in attempting for years to get solanezumab across the FDA finish line — said that it has begun a rolling submission of its rival Alzheimer’s drug donanemab in search of an accelerated approval. Anne White, senior VP of Lilly’s neuroscience unit, acknowledged during the investor call the challenges Biogen has faced with uptake and noted Lilly may face similar hurdles.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

With San­doz con­tin­u­ing to drag on No­var­tis, Vas Narasimhan says he may fi­nal­ly be ready for a sale or spin­off

After years of rehab work aimed at getting Sandoz in fighting trim to compete in a market overshadowed by declining prices, CEO Vas Narasimhan took a big step toward possibly selling or spinning off the giant generic drug player.

The pharma giant flagged plans to launch a strategic review of the business in its Q3 update, noting that “options range from retaining the business to separation.”

Analysts have been poking and prodding Novartis execs for years now as Narasimhan attempted to remodel a business that has been a drag on its performance during most of his reign in the CEO suite. The former R&D chief has made it well known that he’s devoted to the innovative meds side of the business, where they see the greatest potential for growth.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

James Peyer, Cambrian CEO

Brent Saun­ders joins $100M Se­ries C for a com­pa­ny out to be the Bridge­Bio of ag­ing

About a year ago, James Peyer, a CEO and co-founder of the little known longevity biotech Cambrian Biopharma, was trying to find some R&D talent last year when he met with more than a bit of experience in that department: David Nicholson, the former R&D chief of the erstwhile pharma giant Allergan.

It turned out Nicholson already had an interest in Peyer’s field. In their Allergan days, he and COO Brent Saunders held weekly meetups where they tried to figure out how to take the company’s dominance in aesthetics — which, until recently, was often what people meant by anti-aging science — and expertise with more traditional drug development, and use it to make drugs that extend people’s lifespan.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.