Roche touts 2-year ef­fi­ca­cy da­ta for SMA ther­a­py Evrys­di; NIH funds new net­work to study flu and virus­es with ‘pan­dem­ic po­ten­tial’

Two years af­ter ther­a­py, 61% of ba­bies with spinal mus­cu­lar at­ro­phy Type 1 treat­ed with Roche’s Evrys­di (ris­diplam) were able to sit up with­out sup­port for at least five sec­onds com­pared with just 29% of pa­tients af­ter a year, the drug­mak­er said Thurs­day.

As part of the FIRE­FISH-2 Phase III study, re­searchers test­ed Evrys­di’s abil­i­ty to help 41 in­fant pa­tients sit up with­out sup­port at 5 and 30 sec­onds. Da­ta showed that 44% of pa­tients were able to sit with­out sup­port for 30 sec­onds at the two-year check-in com­pared with 17% af­ter one year.

Nine­ty-three per­cent of in­fants were alive af­ter 24 months of treat­ment, Roche said, and 83% per­cent of pa­tients (34/41) were alive and free from per­ma­nent ven­ti­la­tion at that mark. With­out treat­ment, the me­di­an age of death or per­ma­nent ven­ti­la­tion is 13.5 months, Roche said. No new deaths were re­port­ed be­tween months 12 and 24.

On the study’s sec­ondary end­points, Evrys­di al­so main­tained pa­tients’ mo­tor func­tion at two, with 92% of pa­tients be­ing able to feed oral­ly and 95% main­tain­ing the abil­i­ty to swal­low, Roche said. — Kyle Blanken­ship

NIH funds a new net­work to study flu and virus­es with ‘pan­dem­ic po­ten­tial’

Af­ter fund­ing ran out on the NI­AID’s last flu re­search and sur­veil­lance pro­gram, the agency has es­tab­lished and fund­ed a new net­work to track the flu, Covid-19, and oth­er virus­es with pan­dem­ic po­ten­tial.

The new pro­gram, called the Cen­ters of Ex­cel­lence for In­fluen­za Re­search and Re­sponse (CEIRR), in­cludes three of the re­search in­sti­tu­tions in­volved in the last pro­gram, with two ad­di­tions. NI­AID con­tracts will sup­port the net­work for the next sev­en years, with the first year of con­tracts to­tal­ing $24 mil­lion.

The goal is to study and sur­veil the trans­mis­sion of in­fluen­za, in­clud­ing from an­i­mals to hu­mans, to bet­ter un­der­stand how virus­es evolve and adapt. Al­though the pri­ma­ry fo­cus is on the flu, the net­work will al­so study SARS-CoV-2, and oth­er emerg­ing virus­es of con­cern.

CEIRR re­places the for­mer Cen­ters of Ex­cel­lence for In­fluen­za Re­search and Sur­veil­lance (CEIRS) pro­gram, which was fund­ed by con­tracts that ex­pired at the end of last month.

The in­sti­tu­tions award­ed con­tracts this time around in­clude: the Ic­ahn School of Med­i­cine at Mount Sinai for about $6 mil­lion; the Uni­ver­si­ty of Penn­syl­va­nia for near­ly $7 mil­lion; St. Jude Chil­dren’s Re­search Hos­pi­tal for near­ly $9 mil­lion; Emory Uni­ver­si­ty for just over $1 mil­lion; and the Uni­ver­si­ty of Geor­gia Re­search Foun­da­tion for ex­act­ly $1 mil­lion. — Nicole De­Feud­is 

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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UP­DAT­ED: Pfiz­er hits the brakes on their piv­otal tri­al for a BC­MA/CD3 bis­pe­cif­ic on safe­ty con­cerns while FDA road­block is hold­ing up Duchenne MD PhI­II

Pfizer’s ambitious plan to take a Phase II study of its BCMA CD3-targeted bispecific antibody elranatamab (PF-06863135) and run it through to an accelerated approval has derailed.

The pharma giant said in a release this morning that they have halted enrollment for their MagnetisMM-3 study after researchers tracked three cases of peripheral neuropathy in the ongoing Phase I. They are now sharing info with the FDA as they explore the red safety flag.

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FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

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In quest to meet user fee goals, FDA’s per­for­mance con­tin­ues down­ward trend

A recent update to the FDA’s running tally of how it’s meeting its user fee-related performance goals during the pandemic shows an agency that is not out of the woods yet.

The latest numbers reveal that for a second straight quarter in 2021, the FDA has met its user fee goal dates for 93% of original new drug applications, which compares with 94% and 98% for the previous two quarters in 2020, respectively.

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Cynthia Butitta (L) and Joe Jimenez

Is that an­oth­er IPO in the mak­ing? Ex-No­var­tis CEO Joe Jimenez and a lead Kite play­er take up new posts at an off-the-shelf ri­val to 2 pi­o­neer­ing drugs

Right on the heels of taking on a $160 million crossover round in a likely leap to Nasdaq, Century Therapeutics CEO Lalo Flores is now pushing ahead with the high-profile ex-Novartis chief Joe Jimenez as chairman.

Jimenez’s greatest fame at Novartis was earned for one of its weakest products, as their pioneering personalized CAR-T Kymriah won the honors for the first such drug to make it to the market. Now a host of players, including Century, are barreling in behind the frontrunners with allogeneic rivals that can be created for off-the-shelf use.

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Clo­vis spikes ovar­i­an can­cer com­bo af­ter 'un­der­whelm­ing' ef­fi­ca­cy; Servi­er wins pri­or­i­ty re­view for Tib­so­vo ex­pan­sion

Clovis Oncology updated investors with its first quarter results on Wednesday morning, and among its revelations proved some troubling news for a Bristol Myers Squibb-partnered program.

Pairing its experimental lucitanib drug with Bristol Myers’ Opdivo in an ongoing Phase Ib/II study, Clovis reported that “underwhelming efficacy” has led them to cease development for the combo in non-clear-cell ovarian cancer, execs said in Wednesday morning’s earnings call. Enrollment is continuing in other expansion cohorts, which include non-clear cell endometrial cancer, cervical cancer and clear-cell ovarian and endometrial cancers.

Joe Wiley, Amryt CEO

A biotech with a yen for pricey rare dis­ease drugs — and bar­gain base­ment shop­ping — adopts an­oth­er or­phan in lat­est M&A pact

After making it through a long, painful haul to get past a CRL and on to an FDA approval last summer, little Chiasma has found a buyer.

Amryt $AMYT, a company known for its appetite for acquiring expensive drugs for rare diseases at bargain prices, snagged Chiasma and its acromegaly drug Mycapssa (octreotide) capsules in an all-stock deal — with an exchange of 0.396 shares of Amryt for every share of Chiasma.

Ngozi Okonjo-Iweala, Director general of WTO (AP Photo/Keystone/Alessandro Della Bella)

Opin­ion: Waiv­ing Covid-19 vac­cine IP could save lives, but where is the man­u­fac­tur­ing ca­pac­i­ty?

Droves of House Democrats in Washington and members of the European Parliament have now glommed onto a major push by India and South Africa at the WTO to abolish all IP around Covid-19 vaccines.

At first blush, waiving this IP sounds like an easy win: More Covid-19 vaccines made locally for more people means more lives saved. Simple enough, especially as low-income countries have received just a tiny fraction of the world’s vaccine allotment so far.