Poised to launch a new drug for SMA, Bio­gen and Io­n­is shares jump on more promis­ing PhI­II da­ta

Michael Ehlers, Bio­gen

Shares of Bio­gen $BI­IB and Io­n­is Phar­ma­ceu­ti­cals $IONS both jumped ear­ly Mon­day morn­ing af­ter the two biotech part­ners an­nounced an­oth­er pre­lim­i­nary win in a late-stage study of nusin­ersen for spinal mus­cu­lar at­ro­phy.

The lat­est batch of good news — bad­ly need­ed by both of these com­pa­nies — cen­ters on a pos­i­tive read­out for nusin­ersen (Spin­raza) for late-on­set SMA. The first round of promis­ing re­sults dropped three months ago for ear­ly-on­set cas­es and Bio­gen says the FDA has al­ready sig­naled that it plans to act ear­ly on its ap­pli­ca­tion to start sell­ing the drug.

As a re­sult of the pre­lim­i­nary Phase III suc­cess, in­ves­ti­ga­tors are stop­ping the tri­al ear­ly and trans­fer­ring pa­tients over to the ex­tend­ed study. Bio­gen shares surged 6% and Io­n­is stock jumped 18% in pre-mar­ket trad­ing.

Us­ing the Ham­mer­smith Func­tion­al Mo­tor Scale Ex­pand­ed re­port card for pa­tients’ mo­tor func­tions, the drug arm wit­nessed a mean 4.0 im­prove­ment on scores while the place­bo arm dropped by 1.9 points, a 5.9 point over­all spread in the drug’s fa­vor.

That amounts to a “high­ly sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment,” in­ves­ti­ga­tors re­port­ed, which fur­ther backs up their ex­pec­ta­tions for win­ning a near-term ap­proval to sell the drug. Bio­gen says it ex­pects to get a green light from the FDA to start mar­ket­ing the drug ei­ther lat­er this quar­ter or in ear­ly 2017.

That’s wel­come news for Bio­gen in­vestors, who have been deal­ing with a va­ri­ety of set­backs over the course of the year as its Tec­fidera fran­chise be­gins to wane. Back in May, Io­n­is was sent reel­ing af­ter it re­vealed that ev­i­dence of throm­bo­cy­tope­nia—se­vere platelet de­clines — had de­railed one of its late-stage ef­forts. That makes this suc­cess par­tic­u­lar­ly crit­i­cal. And com­ing ear­ly with the sec­ond round of Phase III da­ta didn’t hurt at all.

“The tim­ing and re­sult ma­te­ri­al­ly in­creas­es the as­sur­ance that the ini­tial la­bel for nusin­ersen will in­clude all, or at least most forms of SMA and thus gives the drug even more near term rev­enue po­ten­tial than we have mod­eled pre­vi­ous­ly,” not­ed Leerink’s Ge­of­frey Porges.

Said Michael Ehlers, MD, PhD, ex­ec­u­tive vice pres­i­dent, head of re­search and sevel­op­ment at Bio­gen:

We will make reg­u­la­tors around the globe aware of this da­ta and will con­tin­ue work­ing close­ly with them to bring Spin­raza to fam­i­lies af­fect­ed by SMA as quick­ly as pos­si­ble.

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

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So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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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.

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Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: searching the pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

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Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

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Bris­tol My­ers Squib­b's just-launched MS drug Zeposia makes the cut in key ul­cer­a­tive col­i­tis tri­al

In March, Zeposia became the third oral S1P modulator to secure US approval for multiple sclerosis. Now, the drug has succeeded in a key ulcerative colitis study.

The immunomodulator, akin to others in its class, controls lymphocyte trafficking by limiting the white blood cells to the lymphatic system, in the lymph nodes, and thwarting their ability to jam up lymph nodes — precluding their ability to penetrate the bloodstream and the central nervous system.

Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

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Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

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Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

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Novus Ther­a­peu­tics plunges deep in­to pen­ny stock ter­ri­to­ry af­ter failed ear tri­al

After a more than 15-year run, a California-based biotech is exploring options, including a sale, after its lead experimental therapy failed an exploratory mid-stage study in patients with middle ear infections characterized by a build-up of fluid behind the eardrum.

The company, initially called Tokai Pharmaceuticals but which subsequently changed its name to Novus Therapeutics in 2017, saw its shares more than halve on Monday after the drug — OP0201— did not pass muster as an adjunct therapy to oral antibiotics in infants and children aged 6 to 24 months with acute otitis media (OM).