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.

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

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Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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As­traZeneca gains EU nod for di­a­betes triple; Am­gen and Duke launch re­al-world PC­SK9 ob­ser­va­tion­al study

→ Weeks after winning EU approval to start marketing dapagliflozin as Forxiga, AstraZeneca has racked up another OK for a triplet combo involving the SGLT2 diabetes drug. Named Qtrilmet, the pill combines Forxiga with the DPP-4 inhibitor Onglyza (saxagliptin) and the bedrock drug metformin in a modified-release format. That 3-in-1 approach proved superior in reducing average blood glucose levels to a number of other dual combinations across 5 Phase III trials, including Forxiga plus metformin, Onglyza with metformin, or glimepiride with metformin.

Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is custom, an EMA panel on Friday issued its weekly recommendations on marketing applications submitted by drug developers. This week, the agency backed the use of five new therapies — including two Novartis drugs — but issued no negative reviews.

Novartis’ S1P drug for relapsing forms of multiple sclerosis (MS) drug, Mayzent (known chemically as siponimod), which was approved by the FDA in March — has been given the nod by the EMA. The Swiss drugmaker already sells its other MS drug, Gilenya, in both regions.

Atom­wise's X-37 spin­out gets $14.5 mil­lion to launch AI dis­cov­ery ef­forts

The folks behind Atomwise’s spinout X-37 like to think in cosmological metaphors, and you can think of their AI drug development model as probes sent into space from a central station. That station just got $14.5 million in Series A funding from DCVC Bio, Alpha Intelligence Capital and Hemi Ventures to back those missions.

X-37 uses Atomwise’s AI platform to identify drug targets and – unlike the parent company, which largely sticks to computers  – bring those into a wet lab and preclinical testing.  In addition to AI professionals, it’s led in by part by drug developers from Velocity Pharmaceutical Development.

Ab­bott Lab­o­ra­to­ries CEO Miles White pass­es ba­ton down to suc­ces­sor; Lon­za CEO Marc Funk hits the ex­it

→ Abbott Laboratories has named a successor to CEO Miles White after he announced that he was stepping down in March after 21 years of service. Robert Ford, the company’s COO and president, will take the helm. Ford is known for his work in the $25 billion merger between St. Jude Medical into Abbott in January 2017. White will remain with the company as executive chairman of the board. 

→ After snapping up Novartis’ Swiss facility, Novartis Center of Excellence, in July, Lonza has announced that their CEO, Marc Funk, is hitting the exit for “personal reasons.” Funk has been the CEO of the company for less than a year — brought onto the company back in March. In the meantime, chairman Albert Baehny will serve as interim CEO. 

UCB adds on more pos­i­tive PhI­II da­ta for IL-17A/17F in­hibitor bimek­izum­ab, clear­ing a path to the FDA

A month after posting positive top-line data from their first Phase III trial of the IL-17A/17F inhibitor bimekizumab, Belgium’s UCB says they’ve added more upbeat results from their second late-stage test in moderate-to-severe plaque psoriasis.

That leaves the company on track for regulatory submissions in the middle of next year, says CMO Iris Loew-Friedrich.
Their drug beat out a placebo on the co-primaries — a 90% improvement in PASI 90 (the Psoriasis Area and Severity Index) and Investigator Global Assessment (IGA) response of clear or almost clear (IGA 0/1) at week 16, compared to placebo. Investigators also boasted of hitting some key secondaries.
UCB is angling to enter an increasingly crowded market space.
In their first of 3 Phase III studies for bimekizumab, researchers touted top-line wins on statistically significant results on clearing plaque psoriasis, including a victory over J&J’s IL-23 contender Stelara on key endpoints. The drug targets both IL-17A and IL-17F, a modification on the IL-17A strategy laid out for Taltz (Eli Lilly) and Cosentyx (Novartis). And the new group also includes J&J’s Tremfya and AbbVie’s Skyrizi.

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