Eric Messner, Emalex Biosciences CBO

A Chica­go biotech skips the IPO in fa­vor of late-stage Se­ries D for a PhI­II Tourette drug

Emalex Bio­sciences has se­cured $250 mil­lion in Se­ries D funds to bankroll a Phase III clin­i­cal tri­al in chil­dren, ado­les­cents and adults who have Tourette syn­drome.

The Chica­go biotech plans to kick off the tri­al this quar­ter and will tar­get 90 sites across the US, Cana­da and Eu­rope with a 220-pa­tient tri­al, which the four-year-old drug de­vel­op­er claims would be the largest transat­lantic tri­al ever for the cen­tral ner­vous sys­tem dis­or­der, chief busi­ness of­fi­cer Er­ic Mess­ner told End­points News.

Pump­ing cap­i­tal in­to the busi­ness are: Se­ries D leader Bain Cap­i­tal Life Sci­ences, Val­or Eq­ui­ty Part­ners, Fi­deli­ty Man­age­ment and undis­closed fam­i­ly of­fices.

The tri­al will take about two years, which means da­ta should come out in ear­ly 2025 for a po­ten­tial FDA drug ap­proval re­quest, Mess­ner said. The hefty late-stage pri­vate fi­nanc­ing an­nounced Thurs­day will take Emalex all the way there, the ex­ec­u­tive added, with enough to bankroll man­u­fac­tur­ing, clin­i­cal phar­ma­col­o­gy stud­ies and cer­tain pre-com­mer­cial­iza­tion ac­tiv­i­ties, should the Phase III bear fruit.

Drug R&D for Tourette syn­drome has not been easy, with clin­i­cal set­backs at Neu­ro­crine, Te­va and oth­ers. But some are try­ing to move for­ward, like Noe­ma Phar­ma, which is in mid-stage test­ing for a for­mer Roche as­set.

“Tourette’s is a dis­ease of high un­met need. In the last few years, there have been at­tempts by oth­er in­dus­try lead­ers like Neu­ro­crine Bio­sciences with their VMAT2 in­hibitor, but un­for­tu­nate­ly those tri­als were not suc­cess­ful,” Bain man­ag­ing di­rec­tor Ricky Sun, who led the firm’s in­vest­ment in Emalex, said in an email to End­points. “We be­lieve Emalex’s lead mol­e­cule has the right prop­er­ties to be a high­ly at­trac­tive op­tion for pa­tients.”

Emalex will test the same pri­ma­ry ef­fi­ca­cy end­point as it did in a Phase IIb study of ecopi­pam, or EBS-101, fo­cused on chil­dren un­der the age of 18. The D1AMOND Study test­ed whether 2mg/kg/day of the drug was bet­ter than place­bo on a scale known as the Yale Tic Sever­i­ty Score-To­tal Tic Score in a ran­dom­ized with­draw­al tri­al.

The 153-pa­tient study found that the drug was sta­tis­ti­cal­ly sig­nif­i­cant and clin­i­cal mean­ing­ful “at all time­points, from Week 4 to Week 12,” the com­pa­ny said last No­vem­ber. Headache, fa­tigue, som­no­lence, in­som­nia and rest­less­ness were re­port­ed side ef­fects.

The dopamine-1 re­cep­tor an­tag­o­nist was ac­quired from Mary­land-based Psyadon in 2018. The orig­i­nal own­er had test­ed it in a Phase IIb, with re­sults post­ed in 2017. The drug might al­so be test­ed in pa­tients with rest­less legs syn­drome with aug­men­ta­tion, al­so known as Willis-Ek­bom dis­ease, af­ter a li­cens­ing agree­ment was inked with East Car­oli­na Uni­ver­si­ty last spring.

Mess­ner said the com­pa­ny ex­plored mul­ti­ple fi­nanc­ing av­enues be­tween the $35 mil­lion Se­ries C in March 2021 and the Se­ries D, which came to­geth­er af­ter dis­cus­sions with Bain over the past few months. That in­clud­ed look­ing at a po­ten­tial de­but on Wall Street, the ex­ec­u­tive said.

Cas­tle Creek Bio­sciences, an­oth­er biotech out of the in­cu­ba­tor Paragon Bio­sciences, had al­so con­sid­ered an IPO, but a lit­tle bit more se­ri­ous­ly, hav­ing filed pa­per­work with the SEC but ul­ti­mate­ly back­ing out in late De­cem­ber 2021, in­stead opt­ing for a $112 mil­lion raise in the first half of 2022.

Jeff Aronin

Paragon leader Jeff Aronin and Mess­ner co-found­ed Emalex in 2018. The two worked to­geth­er on Marathon Phar­ma­ceu­ti­cals, a Mar­tin Shkre­li-like price-hike biotech that took a cheap over­seas steroid, got it ap­proved by the FDA for Duchenne mus­cu­lar dy­s­tro­phy in Feb­ru­ary 2017, and re­ceived push­back from politi­cians and oth­ers about the $89,000 list price.

At the time, Mess­ner was VP of sales and mar­ket­ing. PTC Ther­a­peu­tics bought the drug, mar­ket­ed as Em­flaza.

Chica­go-based Paragon has al­so backed day­time sleepi­ness drug­mak­er Har­mo­ny Bio­sciences, Take­da-part­nered Evozyne and eye-fo­cused cell ther­a­py mak­er CiRC Bio­sciences.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Sanofi CFO Jean-Baptiste de Chatillon (L) and CEO Paul Hudson (Romuald Meigneux/Sipa via AP Images)

Sanofi sees downtick in flu sales as it preps for launch of RSV an­ti­body

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

Christophe Weber, Takeda CEO (Photographer: Shoko Takayasu/Bloomberg via Getty Images)

Take­da fo­cus­es on ‘di­verse’ pipeline prospects on heels of two ac­qui­si­tions

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.