Build­ing up its rare obe­si­ty pipeline, Rhythm Phar­ma to raise $150M

With fresh da­ta in-hand for a slew of late-stage clin­i­cal tri­als in rare obe­si­ty dis­eases, Rhythm Phar­ma $RYTM sig­naled plans to­day to raise $150 mil­lion in a fol­low-on of­fer­ing.

Al­though in­vestors aren’t thrilled with the di­lu­tion (an of­fer­ing of this size will make a dent, con­sid­er­ing Rhythm’s mar­ket cap is on­ly $850 mil­lion), the cash sets the com­pa­ny up for years of R&D, clin­i­cal work, and com­mer­cial­iza­tion.

Kei­th Gottes­di­ener

Just yes­ter­day, the com­pa­ny re­port­ed out up­dat­ed da­ta on its lead drug set­melan­otide, a melanocortin-4 re­cep­tor (MC4R) ag­o­nist be­ing test­ed against sev­er­al rare obe­si­ty dis­eases. The com­pa­ny’s most ad­vanced pro­grams are in POMC de­fi­cien­cy obe­si­ty and LepR de­fi­cien­cy obe­si­ty — both of which are in Phase III tri­als. Rhythm’s SEC fil­ing not­ed that $42 mil­lion of the com­pa­ny’s to­tal raise is planned for these two in­di­ca­tions, tak­ing both through FDA ap­proval and com­mer­cial­iza­tion.

Rhythm’s al­so shared up­dat­ed da­ta from the very few pa­tients be­ing stud­ied in the Bardet-Biedl Syn­drome tri­al. Back in Oc­to­ber, Rhythm an­nounced proof-of-con­cept in BBS from five pa­tients. Since then, all four pa­tients who were cat­e­go­rized as weight loss re­spon­ders in the ini­tial read­out con­tin­ue to lose weight. At 46-60 weeks, pa­tients achieved an av­er­age weight loss of 18.5% and an av­er­age hunger score de­crease of 74.2%. Four ad­di­tion­al pa­tients were en­rolled in the study and have been on the treat­ment for “a short du­ra­tion.” Two are show­ing promis­ing re­spons­es, the third is too ear­ly to tell, and the fourth showed no im­prove­ment, the com­pa­ny said.

Rhythm al­so re­port­ed that it’s achieved proof-of-con­cept in Al­ström syn­drome, a rare au­to­so­mal re­ces­sive dis­ease in which child­hood obe­si­ty oc­curs. Again, the tri­al in­volved very few pa­tients. The first pa­tient ex­pe­ri­enced weight loss of 23.2% and a 45.5% de­crease in hunger score af­ter 38 weeks of treat­ment with set­melan­otide. Three more pa­tients were en­rolled in the study and have been on the drug for a short pe­ri­od. One is show­ing a good re­sponse, one is too ear­ly, and the third didn’t show any im­prove­ment.

About $10 mil­lion from the com­pa­ny’s new of­fer­ing is ded­i­cat­ed to push­ing for­ward the BBS and Al­ström pro­grams to Phase III tri­als.

The re­main­ing cash will go to­ward R&D, the rest of the com­pa­ny’s pipeline, and gen­er­al cor­po­rate pur­pos­es.

Kei­th Gottes­di­ener, the com­pa­ny’s CEO, had this to say in a state­ment:

These new clin­i­cal da­ta build up­on pri­or clin­i­cal ex­pe­ri­ence and re­in­force our con­fi­dence in set­melan­otide as a po­ten­tial­ly trans­for­ma­tive treat­ment op­tion. With these re­sults in hand, we are ad­vanc­ing our broad clin­i­cal de­vel­op­ment pro­gram for set­melan­otide with op­ti­mism. We are par­tic­u­lar­ly pleased to progress set­melan­otide in­to a com­bined Phase III pro­gram in BBS and Al­ström Syn­drome, which joins our ful­ly-en­rolled POMC and lep­tin re­cep­tor (LEPR) de­fi­cien­cy obe­si­ty tri­als as our third piv­otal-stage tri­al. Based on re­cent in­ter­ac­tions with the U.S. Food and Drug Ad­min­is­tra­tion (FDA), we have guid­ance for a com­bined tri­al in BBS and Al­ström Syn­drome. This will en­able us to eval­u­ate set­melan­otide si­mul­ta­ne­ous­ly in these two rare ge­net­ic dis­or­ders of obe­si­ty that we be­lieve share a sim­i­lar patho­phys­i­ol­o­gy and clin­i­cal pre­sen­ta­tion, which could po­ten­tial­ly en­able a more rapid path to reg­u­la­to­ry fil­ing.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

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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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

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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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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