Pro­QR spot­lights the most en­cour­ag­ing re­sults from a mixed snap­shot of ef­fi­ca­cy for cys­tic fi­bro­sis

Ex­ecs at Pro­QR Ther­a­peu­tics $PRQR are do­ing their lev­el best this af­ter­noon to ac­cen­tu­ate the pos­i­tive in their ear­ly snap­shot of the im­pact its lead RNA ther­a­py has on cys­tic fi­bro­sis. The big ques­tion is whether they can get in­vestors to look past a slate of failed p-val­ues in the Phase Ib — as well as the im­pli­ca­tions of some heavy­weight com­pe­ti­tion — as they line up reg­u­la­to­ry in­sights for the next big clin­i­cal de­vel­op­ment step.

To be sure, the per pro­to­col da­ta be­ing trot­ted out this af­ter­noon is di­vid­ed among some tiny groups of pa­tients divvied up be­tween 4 dos­ing co­horts and a place­bo arm. In the break­down, QR-010 did achieve sta­tis­ti­cal sig­nif­i­cance for two of the over­all co­horts in mea­sur­ing the re­spons­es on symp­toms from a pa­tient ques­tion­naire. But it al­so missed on the low and high dos­es.

The big is­sue will be FEV1, a cru­cial mea­sure of lung func­tion which is crit­i­cal in cys­tic fi­bro­sis stud­ies. On the ba­sis of FEV1, the drug missed 4 straight shots at sta­tis­ti­cal sig­nif­i­cance in tiny dos­ing groups of 5 or 6 pa­tients, of­ten bad­ly. The high 50 mg dose tracked close to the place­bo arm, with 8 pa­tients. On­ly by mov­ing to a sub­group of pa­tients with low­er lung func­tion scores did re­searchers find one dose that hit the FEV1 mark with a 10.9% im­prove­ment.

En­cour­ag­ing­ly, the same 12.5 mg dose arm in the sub­group al­so had the best re­sponse on symp­toms: plus 27.5 com­pared to an 11.8 point drop in the place­bo arm.

These da­ta takes some ex­plain­ing, CEO and com­pa­ny founder Daniel de Boer tells me.

The pri­ma­ry goals are safe­ty and tol­er­a­bil­i­ty, he says, with a shot at some ex­plorato­ry end­points. In the up­com­ing Phase II study, they’ll move away from a pop­u­la­tion of pa­tients with near nor­mal lev­els of lung func­tion — where it can be very dif­fi­cult to track im­prove­ments of any kind — in­to pa­tients where they saw the best re­spons­es in this small study.

“We’re ex­cit­ed,” says de Boer, who got start­ed with the help of the late Hen­ri Ter­meer. There was plen­ty of clin­i­cal­ly mean­ing­ful da­ta in the study to in­di­cate the drug’s po­ten­tial. And while the press will be on to meet with reg­u­la­tors on next steps, the Dutch biotech al­so plans to make sure they can move on Phase II rel­a­tive­ly quick­ly, as ri­vals in the field add pres­sure to per­form well as the land­scape shifts.

Un­like the leader in CF — Ver­tex, which is pitch­ing a com­bi­na­tion of teza­caftor/iva­caftor that tar­gets the F508del mu­ta­tion to get the CFTR pro­tein to the cell sur­face, where an­oth­er drug amps its per­for­mance — Pro­QR tries to achieve its goal with an in­haled ther­a­py that binds specif­i­cal­ly to the de­fec­tive CFTR RNA in or­der to cor­rect the F508del mu­ta­tion and re­store the func­tion of the CFTR pro­tein, stop­ping dis­ease pro­gres­sion.

Ver­tex has had some stel­lar da­ta to re­port to reg­u­la­tors, and ex­pe­ri­ence in a field that has de­liv­ered block­buster ex­pec­ta­tions about its fu­ture. But de Boer says that he ex­pects that ul­ti­mate­ly there will be a va­ri­ety of drugs on the CF mar­ket, with doc­tors and pa­tients able to pick the one that will work best for them.

That’s a field he plans to be com­pet­i­tive in — pro­vid­ed the next round of ef­fi­ca­cy da­ta starts to look dra­mat­i­cal­ly bet­ter.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

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Mickey Kertesz, KidsandArtOrg via YouTube

Soft­Bank's newest, $165M biotech in­vest­ment looks for in­fec­tious traces in the blood

SoftBank has found its newest biotech investment.

The Japanese bank has invested $165 million into Karius, a company that uses blood tests to diagnose infectious diseases, as part of its new Vision Fund 2. The full scope of the new fund has yet to be announced, but the first and newly-beleaguered Vision Fund poured $100 billion into technology companies, including the biotechs Vir Biotechnology and Roivant and the sequencing company 10x Genomics.

Methicillin-resistant Staph aureus (Shutterstock)

FDA grants ‘break­through’ sta­tus to an­tibi­ot­ic al­ter­na­tive as Con­tra­Fect rush­es to join fight against su­per­bug

An experimental drug that promises to be the first anti-infective agent to prove superior to vancomycin — an antibiotic approved in 1958 — has notched the FDA’s “breakthrough” status.

ContraFect said the designation was based on Phase II data in which exebacase was tested against a superbug known as methicillin-resistant Staph aureus, or MRSA. In a subgroup analysis, the clinical responder rate at day 14 was 42.8% higher than that among those treated with standard of care, the company said (p=0.010).

Zhong Nanshan, CGTN via YouTube

Har­vard joins coro­n­avirus fight with $115 mil­lion and a high-pro­file Chi­nese part­ner

For two months, as the novel coronavirus swelled from a few early cases tied to a Wuhan market to a global epidemic, most of the world’s focus and dollars have flowed toward emergency initiatives: building vaccines at a record pace, plucking experimental antivirals out of freezers to see what sticks and immunizing mice for new antibodies.

Now a new and well-funded collaboration between Harvard and a top Chinese research institute will play the long game. In a 5-year, $115 million initiative backed by China Evergrande Group, researchers from the Harvard Medical School, Harvard T.H. Chan School of Public Health and Guangzhou Institute for Respiratory Health will study the virus in an effort to develop therapies against infections by the novel coronavirus, known as SARS–CoV-2, and to prevent new ones.

No­var­tis gets a boost in block­buster mul­ti­ple scle­ro­sis race with Roche

In the first step of what’s likely to be a long and uphill battle for the drugmaker, the FDA has accepted Novartis’s BLA submission for a new multiple sclerosis drug and given it priority review. The PDUFA date for the potential blockbuster drug is in June.

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Juergen Horn

An­i­mal health vet Juer­gen Horn makes new an­ti­body play for pets, rak­ing $15M in Se­ries A haul

Zoetis forked over $85 million in 2017 to acquire Nexvet Biopharma and its pipeline of monoclonal antibodies. Juergen Horn, Nexvet’s former chief product development officer, has now secured $15 million for his own biologic company for animals: Invetx.

Buoyed by emerging advances in gene therapies for humans, scientists have started looking at harnessing the technology for animals setting up companies such as Penn-partnered Scout Bio and George Church-founded Rejuvenate Bio. But akin to Nexvet, Invetx is working on leveraging the time-tested science of monoclonal antibodies to treat chronic diseases that afflict man’s best friend.

As coro­n­avirus out­break reach­es 'tip­ping point,' GSK lends ad­ju­vant tech to Chi­nese part­ner armed with pre­clin­i­cal vac­cine

As the coronavirus originating out of Wuhan spreads to South Korea, Italy and Iran, stoking already intense fears of a pandemic, GlaxoSmithKline has found another pair of trusted hands to place its adjuvant system. China’s Clover Biopharmaceuticals will add the adjuvant to its preclinical, protein-based vaccine candidate against SARS-CoV-2.

Clover, which is based in the inland city of Chengdu, boasts of a platform dubbed Trimer-Tag that produces covalently-trimerized fusion proteins. Its candidate, COVID-19 S-Trimer, resembles the viral spike (S)-protein found in the virus.