Es­pe­ri­on shares surge as FDA waves off pan­el re­view; Adap­ti­m­mune buoyed by a pos­i­tive da­ta snap­shot

Derek Archi­la

→ Es­pe­ri­on got a 15% bump on its stock price $ES­PR this morn­ing af­ter the biotech re­port­ed that the FDA had said it didn’t ex­pect to call an ad­vi­so­ry pan­el to­geth­er to re­view the new­ly ac­cept­ed NDAs for be­mpe­doic acid (BPA) and their be­mpe­doic acid/eze­tim­ibe com­bi­na­tion tablet. In­vestors saw that as a pos­i­tive sign for the LDL-low­er­ing ther­a­py. The PDU­FA date is Feb­ru­ary 21. The lack of an ad-com is an “im­por­tant, in­cre­men­tal pos­i­tive giv­en the in­vestor con­tro­ver­sies sur­round­ing BPA’s safe­ty and the as­sump­tion among most in­vestors that an ad­com was a fore­gone con­clu­sion; how­ev­er we would note, the FDA could still con­vene an ad­com if it feels one is need­ed at any point dur­ing the re­view process,” Stifel‘s Derek Archi­la wrote in a note.

James No­ble

Shares of Adap­ti­m­mune perked up Mon­day af­ter the biotech $ADAP re­port­ed out a snap­shot of ear­ly pos­i­tive da­ta from their ADP-A2M4 pi­lot study. Re­searchers for the UK/US biotech say the T cell ther­a­py kicked up a re­sponse in 4 out of 5 syn­ovial sar­co­ma pa­tients. CEO James No­ble tout­ed the da­ta, say­ing they were now ready to launch their SPEAR­HEAD-1 tri­al in pa­tients with syn­ovial sar­co­ma and myx­oid/round cell li­posar­co­ma this year, putting them on track for a mar­ket launch slat­ed for 2022. Shares were up 13% in morn­ing trad­ing.

Liz Bar­rett

Af­ter poach­ing No­var­tis $NVS on­col­o­gy group head Liz Bar­rett in­to tak­ing charge of the small biotech, Uro­Gen $URGN in Jan­u­ary re­port­ed that its lead drug, UGN-101, scored in a late-stage study in pa­tients with low-grade up­per tract urothe­lial can­cer. Over the week­end, Uro­Gen pre­sent­ed a sec­ondary analy­sis of the tri­al, in which 71 pa­tients were treat­ed and all have un­der­gone pri­ma­ry dis­ease eval­u­a­tion. The com­plete re­sponse (CR) rate was 59% — con­sis­tent with Uro­gen’s Jan­u­ary pre­sen­ta­tion — and well above what the com­pa­ny and our con­sul­tants have pre­vi­ous­ly in­di­cat­ed would be nec­es­sary to sup­port ap­proval (~20% CR), Cowen‘s Boris Peak­er wrote in a note. “The…up­date in­di­cates that none of the pa­tients who were ini­tial­ly re­sectable had dis­ease re­cur­rence with­in 6 months, a key da­ta point in es­tab­lish­ing UGN-101 as an al­ter­na­tive to surgery. Based on the re­sults to date, we an­tic­i­pate that UGN-101 will show fa­vor­able dura­bil­i­ty com­pared to avail­able op­tions.”

Savara‘s rare lung dis­ease drug Mol­gradex has won the FDA’s fast track sta­tus. The drug is the com­pa­ny’s lead ex­per­i­men­tal treat­ment and is be­ing eval­u­at­ed in piv­otal study in pa­tients with the treat­ment of au­toim­mune pul­monary alve­o­lar pro­teinosis (aPAP), for which topline re­sults are ex­pect­ed in June.

Af­ter Reg­u­lus Ther­a­peu­tics $RGLS pulled off a re­verse split of its stock, and of­floaded its lead drug to its part­ners at Sanofi $SNY last year, the trou­bled La Jol­la-based com­pa­ny has amend­ed its loan with Ox­ford Fi­nance LLC. It pro­vides Reg­u­lus with a new twelve-month pe­ri­od of in­ter­est-on­ly pay­ments, com­menc­ing May 2019 and a two-year ex­ten­sion of its ma­tu­ri­ty date to May 2022.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.