Spin­raza ri­val? With DMD flops in the rearview mir­ror, PTC Ther­a­peu­tics posts strong SMA da­ta

Af­ter years try­ing to push a thrice-failed Duchenne drug in­to the US mar­ket (and ul­ti­mate­ly fail­ing to do so), PTC Ther­a­peu­tics is fi­nal­ly on­to some­thing in­ter­est­ing.

Gio­van­ni Baranel­lo

The New Jer­sey com­pa­ny’s stock $PTCT is soar­ing on news of a com­plete­ly dif­fer­ent pro­gram — one part­nered with Roche — that’s just im­pressed an­a­lysts and in­vestors with up­dat­ed da­ta from a Phase I tri­al.

This drug, called ris­diplam or RG7916, is be­ing test­ed in ba­bies with spinal mus­cu­lar at­ro­phy, a ge­net­ic con­di­tion that robs peo­ple of phys­i­cal strength by af­fect­ing the mo­tor nerve cells in the spinal cord. It’s the num­ber one ge­net­ic cause of death for in­fants. The dis­ease is caused by a gene mu­ta­tion that pre­vents the body from pro­duc­ing a mus­cle de­vel­op­ment pro­tein called SMN, which leads to at­ro­phy of the mus­cles, mo­tor im­pair­ment, and paral­y­sis. Ba­bies born with the sever­est kind of SMA nev­er learn to crawl, sit up, or walk, and many lose the abil­i­ty to breathe. They of­ten die with­in 18 months.

PTC’s drug, li­censed to Roche in 2011, is de­signed to re­place the miss­ing SMN pro­tein — and it can be tak­en oral­ly. The small tri­al test­ed RG7916 in 21 ba­bies born with the sever­est form of SMA. In the up­dat­ed Phase I da­ta, the drug showed to im­prove mo­tor func­tion in pa­tients, with a me­di­an im­prove­ment of 5.5 points in a test known as the CHOP-IN­TEND. This test, de­vel­oped by the Chil­dren’s Hos­pi­tal of Philadel­phia, mea­sures mus­cle de­vel­op­ment mile­stones.

See­ing im­prove­ment in mus­cle func­tion this ear­ly was a bit of a sur­prise — which is why in­vestors are so gid­dy.

“Da­ta on mo­tor func­tion seem more en­cour­ag­ing when we con­sid­er that we are see­ing mo­tor func­tion im­prove­ments and mile­stones achieve­ment at this ear­ly stage of the study, which was es­sen­tial­ly a dose-find­ing study and most of the in­fants in­clud­ed have re­ceived their first dose af­ter the age of 5 months,” said in­ves­ti­ga­tor Gio­van­ni Baranel­lo at the Fon­dazione Is­ti­tu­to Neu­ro­logi­co Car­lo Besta. “It is ex­cit­ing to see ev­i­dence of clin­i­cal ben­e­fit from a sys­temic oral treat­ment for SMA.”

An­a­lysts are posit­ing that RG7916 could one day be com­pet­i­tive to Spin­raza — the block­buster SMA drug de­vel­oped by Io­n­is and li­censed by Bio­gen. Spin­raza, al­so de­signed to boost SMN pro­tein, is de­liv­ered via a shot to the spine. If PTC/Roche’s drug was equal­ly ef­fec­tive but eas­i­er to take via pill, the med­i­cine could be a sol­id ri­val.

It should be not­ed that there’s an­oth­er po­ten­tial Spin­raza ri­val com­ing down the pipeline: a gene ther­a­py de­vel­oped by Avex­is and re­cent­ly bought by No­var­tis in the $8.7 bil­lion Avex­is buy­out in April.

Re­gard­less, the da­ta are good news for PTC, a com­pa­ny that’s been plagued with bad news for years due to the fail­ure (af­ter fail­ure af­ter fail­ure) of its Duchenne mus­cu­lar dy­s­tro­phy drug ataluren. That drug, ap­proved in Eu­rope, has so far failed to please reg­u­la­tors in the US. Al­though PTC still isn’t giv­ing up on DMD, in­vestors are sure­ly hap­py to see the com­pa­ny mov­ing on to green­er pas­tures. The com­pa­ny’s stock is up 23% Mon­day morn­ing, trad­ing at $46.29 as of press time. That’s up from Fri­day’s close of $37.55 per share.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,000+ biopharma pros reading Endpoints daily — and it's free.

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

Endpoints Premium

Premium subscription required

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

Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,000+ biopharma pros reading Endpoints daily — and it's free.

Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,000+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,000+ biopharma pros reading Endpoints daily — and it's free.

Iterum's fu­ture looks un­cer­tain, af­ter lead an­tibi­ot­ic fails con­sec­u­tive piv­otal stud­ies

While the market for antibiotics remains in tatters — unlike many of its bankrupt (or at the brink of bankruptcy) peers — Iterum is suffering not because its antibiotic isn’t selling, but because the compound has now failed back-to-back late-stage studies.

The experimental drug, sulopenem, was designed to tackle drug-resistant infections with an outpatient focus (in addition to hospitals), to avert those reimbursement challenges that incentivize hospitals to prescribe cheaper, generic broad-spectrum antibiotics.

Covid-19 roundup: Did in­sid­ers cash in on pos­i­tive news re­port about Gilead be­fore pub­li­ca­tion?

A series of bullish trades on Gilead options just before the release of a favorable news story is raising questions among regulatory experts, Reuters reported.

On April 16, just hours before STAT published anecdotes from a Chicago hospital that served as one of the clinical sites to test Gilead’s remdesivir in Covid-19 patients, the California-based company’s shares were trading at around $75. Four large blocks of options were purchased for about $1.5 million each, betting that the stock would rise beyond that to as much as $87.5 by mid-August.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,000+ biopharma pros reading Endpoints daily — and it's free.

Roche nabs front­line OK for Avastin/Tecen­triq in com­mon liv­er can­cer, best­ing an old Bay­er drug

For the first time in 12 years, the FDA has approved a new frontline treatment for the most common form of liver cancer.

The agency okayed a combination of Roche’s anti-VEGF antibody Avastin and their immunotherapy Tecentriq for patients with unresectable or metastatic hepatocellular carcinoma (HCC). The approval comes two weeks after Roche and their big biotech sub Genentech published Phase III results showing the combo improved both progression-free survival and, crucially, helped patients live longer than the long-running standard-of-care, Bayer’s Nexavar.