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.

Fangliang Zhang, AP Images

UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

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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