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.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Jackie Fouse, Agios CEO

Agios scores its sec­ond pos­i­tive round of da­ta for its lead pipeline drug — but that won't an­swer the stub­born ques­tions that sur­round this pro­gram

Agios $AGIO bet the farm on its PKR activator drug mitapivat when it recently decided to sell off its pioneering cancer drug Tibsovo and go back to being a development-stage company — for what CEO Jackie Fouse hoped would be a short stretch before they got back into commercialization.

On Tuesday evening, the bellwether biotech flashed more positive topline data — this time from a small group of patients in a single-arm study. And the executive team plans to package this with its earlier positive results from a controlled study to make its case for a quick OK.

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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George Yancopoulos (L) and Len Schleifer (Regeneron)

Re­gen­eron touts pos­i­tive pre­lim­i­nary im­pact of its Covid an­ti­body cock­tail, pre­vent­ing symp­to­matic in­fec­tions in high-risk group

Regeneron flipped its cards on an interim analysis of the data being collected for its Covid-19 antibody cocktail used as a safeguard against exposure to the virus. And the results are distinctly positive.

The big biotech reported Tuesday morning that their casirivimab and imdevimab combo prevented any symptomatic infections from occurring in a group of 186 people exposed to the virus through a family connection, while the placebo arm saw 8 of 223 people experience symptomatic infection. Symptomatic combined with asymptomatic infections occurred in 23 people among the 223 placebo patients compared to 10 of the 186 subjects in the cocktail arm.

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Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Vir's CMO says he's sur­prised that a low dose of their he­pati­tis B drug ap­pears promis­ing in ear­ly slice of da­ta — shares soar

Initial topline data from a Phase I study of a new therapeutic for chronic hepatitis B virus was so promising that it surprised even the CMO of the company that produces it.

Vir Biotechnology on Tuesday announced that its VIR-3434 molecule reduced the level of virus surface antigens present in a blinded patient cohort after eight days of the trial with just a single 6 mg dose. Six of the eight patients in the cohort were given the molecule, and the other two a placebo—all six who received the molecule saw a mean antigen reduction of 1.3 log10 IU/mL, Vir said.

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David Marek, Myovant

My­ovant beefs up da­ta pack­age in NDA #3, boost­ing its case for longterm dos­ing of Pfiz­er-part­nered re­l­u­golix

When Pfizer handed over $650 million in cash to partner on Myovant’s relugolix, the pharma giant made clear that the deal — valued at $4.2 billion total — was just as much about the approved indication of prostate cancer as the two women’s health conditions the drug could treat.

A month later, the two companies are offering another glimpse of the therapy’s longterm potential in endometriosis.

Look­ing to win over some skep­ti­cal an­a­lysts, Rhythm beats the drum on in­ter­im da­ta in PhII bas­ket study for ad­di­tion­al in­di­ca­tions

Rhythm Pharmaceuticals has been working toward expanding the FDA approval they received just two months ago for three rare genetic disorders that result in obesity. In December, their Phase III cut of data saw mixed reactions from analysts, but new interim results released Tuesday may provide more excitement.

In an ongoing Phase II study for setmelanotide across individuals with one of three distinct rare genetic diseases of obesity, 65 patients had reached the Dec. 17 cutoff date for evaluation. Among patients who met the primary endpoint of at least 5% weight loss over three months, Rhythm saw an average reduction of no less than 7.1% in any of the groups.