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.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Mer­ck helps bankroll new part­ner Themis' game plan to fin­ish the chikun­gun­ya race and be­gin on­colyt­ic virus quest

As Themis gears up for a Phase III trial of its chikungunya vaccine, the Vienna-based biotech has closed out €40 million ($44 million) to foot the clinical and manufacturing bills.

Its heavyweight partners at Merck — which signed a pact around a mysterious “blockbuster indication” last month — jumped into the Series D, led by new investors Farallon Capital and Hadean Ventures. Adjuvant Capital also joined, as did current investors Global Health Investment Fund, aws Gruenderfonds, Omnes Capital, Ventech and Wellington Partners Life Sciences.