Ul­tragenyx shares tum­ble low­er af­ter rare dis­ease drug fails PhII tri­al

Emil Kakkis, Ul­tragenyx

Shares of Ul­tragenyx $RARE tum­bled close to 10% af­ter the rare dis­ease biotech re­port­ed that one of its drugs failed a Phase II tri­al.

UX007 was test­ed as a treat­ment for glu­cose trans­porter type-1 de­fi­cien­cy syn­drome—or Glut1 DS—among a small group of pa­tients. But it on­ly cut the rate of seizures in the drug arm by 13.4% com­pared to the place­bo arm, falling short of sta­tis­ti­cal sig­nif­i­cance.

In­ves­ti­ga­tors, though, teased out a clin­i­cal­ly, though not sta­tis­ti­cal­ly, sig­nif­i­cant ben­e­fit for pa­tients ex­pe­ri­enc­ing what are called “ab­sence seizures,” or pe­ri­ods of un­re­spon­sive­ness. And that is what Ul­tragenyx now in­tends to fo­cus on in a Phase III piv­otal tri­al.

The re­searchers said that they tracked a 47.3% drop in ab­sence seizure fre­quen­cy rel­a­tive to the base­line. There was a 9.1% drop in ob­serv­able seizures.

There were no se­ri­ous ad­verse events record­ed in the tri­al, but 14 of 36 pa­tients dropped out due to ad­verse events. 18 pa­tients in the drug arm — 72% — re­port­ed an ad­verse event com­pared to 45% in the place­bo arm.

The drug con­verts in­to glu­cose in the brain, treat­ing a ge­net­ic dis­ease that in­ter­rupts the sup­ply of glu­cose, trig­ger­ing an en­er­gy de­fi­cien­cy with se­vere side ef­fects. There are no ap­proved ther­a­pies for Glut1 DS, which is gen­er­al­ly cur­rent­ly treat­ed with a ke­to­genic di­et.

“These da­ta sug­gest that UX007 has a clin­i­cal­ly mean­ing­ful ef­fect in Glut1DS pa­tients with ab­sence seizures,” said Ul­tragenyx CEO Emil Kakkis in a state­ment. “We look for­ward to study­ing UX007 in our Phase 3 study in Glut1 DS pa­tients with move­ment dis­or­ders, and con­tin­ue to eval­u­ate our plans in the seizure in­di­ca­tion.”

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

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

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

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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

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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)

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After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

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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