Puretech-backed Karuna makes Nas­daq de­but with $89M IPO

Bet­ting on the po­ten­tial of two old­er drugs to fight Alzheimer’s and schiz­o­phre­nia — Boston-based Karuna Ther­a­peu­tics has priced its up­sized ini­tial pub­lic of­fer­ing in the mid­dle of the range, rais­ing gross pro­ceeds of about $89 mil­lion.

The com­pa­ny, which will de­but on the Nas­daq un­der the sym­bol “$KRTX” on Fri­day, is work­ing on the hy­poth­e­sis that a com­bi­na­tion of mus­carinic acetyl­choline re­cep­tor ag­o­nist xanome­line — an Eli Lil­ly $LLY castoff — and tro­spi­um chlo­ride, an FDA-ap­proved mus­carinic re­cep­tor an­tag­o­nist to­geth­er will make an adept an­tipsy­chot­ic.

Steven Paul Linkedin

Once up­on a time, star CNS re­searcher (and for­mer Voy­ager chief) Steven Paul was at Lil­ly, where he worked on xanome­line. The drug is de­signed to tar­get M1 and M4 re­cep­tors in the brain — but ad­verse events trig­gered by off-tar­get ef­fects soured the US drug­mak­er on the drug. Last Au­gust, he was one of the syn­di­cates of in­vestors in­clud­ing ARCH Ven­ture Part­ners and the Well­come Trust that backed Karuna.

Ex­ist­ing an­tipsy­chot­ic treat­ments work pri­mar­i­ly by in­hibit­ing D2 dopamine re­cep­tors. Mus­carinic re­cep­tor ag­o­nists emerged in the 1990s as a po­ten­tial al­ter­na­tive ap­proach for treat­ing psy­chosis —  but the de­vel­op­ment of such ther­a­peu­tics has been lim­it­ed by un­de­sir­able side-ef­fects that are trig­gered by the stim­u­la­tion of mus­carinic re­cep­tors in pe­riph­er­al tis­sues.

An­drew Miller Linkedin

Karuna — whose COO is MIT grad­u­ate An­drew Miller — con­tends that the ad­di­tion of tro­spi­um chlo­ride will counter the ad­verse event pro­file of xanome­line. The com­bined prod­uct,  dubbed KarXT, is de­signed to make a bee­line for mus­carinic re­cep­tors in the cen­tral ner­vous sys­tem and to ig­nore pe­riph­er­al tis­sues.

The lead ex­per­i­men­tal treat­ment is be­ing test­ed as a treat­ment for psy­chosis in a mid-stage study with schiz­o­phre­nia pa­tients — da­ta is ex­pect­ed by the end of the year. It is al­so in ear­ly-stage pro­grams for oth­er schiz­o­phre­nia symp­toms, Alzheimer’s-re­lat­ed psy­chosis and pain.

Oth­er drug­mak­ers have al­so been se­duced by the po­ten­tial of mus­carinic re­cep­tors. For in­stance, Al­ler­gan $AGN inked a $3.3 bil­lion deal to in-li­cense M1 and M4 tar­get­ed drugs from Hep­tares in the spring of 2016.

Less than a year af­ter rais­ing $42 mil­lion, PureTech-backed Karuna laid the ground­work for a $75 mil­lion IPO ear­li­er in June. Karuna orig­i­nal­ly filed to of­fer 4.4 mil­lion shares. On Thurs­day, the firm said it is of­fer­ing 5.6 mil­lion at $16, the mid­point of the $15 to $17 range.

So­cial im­age: Steven Paul, Karuna

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.
Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.

Eli Lil­ly-backed biotech grabs $100M to dis­patch an­ti­body-oligonu­cleotide con­ju­gates af­ter mus­cu­lar dy­s­tro­phy

Hold up your hand. Make a fist. Now open it. And again.

If you can do it fully and with ease, then the proteins in your hand are likely working properly. If you can’t then they may not be. In people with myotonic muscular dystrophy, something more atomic is going on.

In those folks, the problem is RNA. Certain base pairs repeat far beyond normal, up to 11,000 superfluous letters in some cases. The extended strands form “clumps.” Proteins misform and can’t function properly. They often allow one movement but not the reverse, a condition called myotonia that gives the dystrophy its name.

As­traZeneca sets stage for mar­ket­ing ap­pli­ca­tion with promis­ing piv­otal lu­pus drug da­ta

After fumbling in its first late-stage lupus study, AstraZeneca disclosed that a second pivotal trial testing its experimental drug, anifrolumab, had met the main goal, in August. Earlier this week, the British drugmaker broke out the numbers from its successful study.

Last year, anifrolumab failed to meet the main goal of diminishing disease activity in the 460-patient TULIP I study, a 52-week trial that tested two doses of the drug versus a placebo. But in the 373-patient TULIP II study, the higher dose (300 mg) was compared to patients given a placebo — patients in both arms were on baseline standard care.

FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.

The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.

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FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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