Ul­tragenyx shares surge as block­buster hope­ful buro­sum­ab con­tin­ues to per­form in piv­otal study

Emil Kakkis, Ul­tragenyx

Just in time for its R&D day to­day, Uly­tragenyx $RARE and its part­ners at Ky­owa Hakko Kirin out­lined up­dat­ed 48 week da­ta on buro­sum­ab, which is on track to get a like­ly FDA ap­proval in the spring.

Mov­ing the goal line out to 48 weeks, from 24, re­searchers say that 84% of pa­tients suf­fer­ing from X-linked hy­pophos­phatemia — or XLH — and on the drug from the start of the tri­al hit their goal on serum phos­pho­rus lev­els, which was the pri­ma­ry end­point. And 89% of the switchovers from the place­bo group al­so hit their marks on the bio­mark­er for ef­fi­ca­cy.

Two mea­sures of stiff­ness and phys­i­cal func­tion al­so re­flect­ed pos­i­tive re­sults for the drug, along­side an im­prove­ment in frac­ture heal­ing.

The ther­a­py, ini­tial­ly de­vel­oped by Ky­owa Hakko Kirin, is a mon­o­clon­al IgG1 an­ti­body that tar­gets FGF23, a hor­mone linked to the re­duc­tion of serum lev­els of phos­pho­rus and ac­tive vi­t­a­min D.

This drug is cen­tral to Ul­tragenyx’s nar­ra­tive. The biotech just scored an ap­proval for Mep­se­vii, a drug that treats rare cas­es of mu­copolysac­cha­ri­do­sis type VII . But it’s the buro­sum­ab launch af­ter the April 17 PDU­FA date that has every­one’s at­ten­tion on Wall Street. The biotech has been set­ting up a small, 30-per­son sales force as it be­gins to han­dle what Ul­tragenyx ex­ecs hope will be a sub­stan­tial port­fo­lio of rare dis­ease drugs.  And buro­sum­ab has peak sales fig­ures of $1 bil­lion tied to it.

A num­ber of an­a­lysts are look­ing for Ul­tragenyx to use to­day’s pre­sen­ta­tion to in­vestors as a chance to ex­plain in more de­tail how the com­pa­ny plans to lever­age the gene ther­a­py plat­form it ac­quired with the Di­men­sion buy­out ear­li­er in the year.

The biotech’s shares surged 6% on the lat­est da­ta read­out.

“This longer term da­ta on symp­tom im­prove­ment and frac­ture heal­ing sup­port buro­sum­ab’s po­ten­tial val­ue in treat­ing se­ri­ous dis­ease symp­toms and pro­mot­ing bone heal­ing in adult pa­tients with XLH,” said Ul­tragenyx CEO Emil Kakkis in a state­ment. “The con­tin­ued clin­i­cal im­prove­ments in pa­tients and the new da­ta demon­strat­ing a sig­nif­i­cant de­crease in pain med­ica­tion use af­ter treat­ment with buro­sum­ab pro­vide fur­ther sup­port for the po­ten­tial val­ue in the treat­ment of adults with XLH.”

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.

The key dates for KRAS watch­ers through the end of the year — the trail is nar­row and risks are ex­treme

There’s nothing quite like a big patent win when it comes to burnishing your prospects in the pipeline. And for Amgen, which seems to have rescued Enbrel for a run to 2029, the cheering section on Wall Street is now fixed on AMG 510 and a key rival.

And it didn’t take much data to do it. 

There was the first snapshot of a handful of patients, with a 50% response rate. Then came word that Amgen researchers are also tracking responses in different cancers, at least one in colorectal cancer and appendiceal too. 

Bain's Or­ly Mis­han joins Pfiz­er's neu­ro spin­out Cerev­el; On­colyt­ic virus biotech taps Sil­la­Jen ex­ec He­le­na Chaye as CEO

→ Bain Capital is deploying one of its top investors to Cerevel Therapeutics, steering a $350 million-plus neuro play carved out of Pfizer. Orly Mishan — a co-founder and principal of Bain’s life sciences unit — was involved in the partnership that birthed the biotech spinout in the first place. As Cerevel’s first chief business officer, she is tasked with corporate development, program management as well as technical operations. 

UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.