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.”

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Fabrice Chouraqui, Cellarity CEO-partner (LinkedIn)

Drug de­vel­op­er, Big Phar­ma com­mer­cial ex­ec, now an up­start biotech chief — Fab­rice Chouraqui is ready to try some­thing new as a ‘CEO-part­ner’ at Flag­ship

Fabrice Chouraqui’s career has taken some big twists along his life journey. He got his PharmD at Université Paris Descartes and jumped into the drug development game for a bit. Then he took a sharp turn and went back to school to get his MBA at Insead before returning to pharma on the commercial side.

Twenty years later, after steadily rising through the ranks and journeying the globe to nab a top job as president of US pharma for the Basel-based Novartis, Chouraqui exited in another career switch. And now he’s headed into a hybrid position as a CEO-partner at Flagship, where he’ll take a shot at leading Cellarity — one of the VC’s latest paradigm-changing companies of the groundbreaking model that aspires to deliver a new platform to the world of drug R&D.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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As­traZeneca’s $7B ADC suc­ceeds where Roche failed, im­prov­ing sur­vival in gas­tric can­cer

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which they’ll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.

Once a gem, now just a rock, Take­da punts PhI­II IBD drug as ri­vals mus­cle ahead

Back in 2016, when then-Shire CEO Flemming Ørnskov picked up a promising clinical-stage IBD drug from Pfizer, the Boston-based biotech dubbed it SHP647 and moved it into the gem section of the pipeline, with rosy expectations of registration-worthy Phase III data ahead.

This was a drug that the EC wanted Takeda to commit to selling off before it gave their blessing to its acquisition of Shire, to settle some deep-seated concerns revolving around the potential market overlap with their blockbuster rival Entyvio. And Takeda, which took on a heavy debt load to buy Shire, clearly wanted the cash to pay down debt.

Ear­ly sur­vival da­ta boost Zio­phar­m's 'con­trolled IL-12' im­munother­a­py for glioblas­toma

An unconventional pairing of a gene therapy and an oral drug that promises to attack recurrent or progressive glioblastoma with controlled release of IL-12 has turned up more promising — if early — overall survival data. On top of boosting its case as a monotherapy, the data can also bode well for a combination with Regeneron’s PD-1 inhibitor, Libtayo.

Both the treatment and its developer, Ziopharm Oncology, have come a long way. The stock price peaked in 2015 but cratered in 2016 following a patient death in a Phase I.