A Cana­di­an start­up re­fu­els to take on a rare car­diac con­di­tion that many pa­tients don't re­al­ize they have

A year and a half ago, a small biotech launched out of Laval, Que­bec to one-up be­ta block­ers for the treat­ment of a rare heart con­di­tion that of­ten goes un­di­ag­nosed. Now, a slate of in­vestors is reach­ing deep­er in­to the pock­ets to dri­ve the com­pa­ny’s push to­ward the clin­ic.

LQT Ther­a­peu­tics un­veiled a $19 mil­lion Se­ries A round on Tues­day to bring its lead com­pound for Long QT syn­drome (LQTS) through Phase I. LQTS is a ge­net­ic dis­or­der that caus­es an elon­ga­tion be­tween the Q and T waves dur­ing a heart­beat, which can lead to rapid, chaot­ic heart­beats or ar­rhyth­mias. Pa­tients with the con­di­tion suf­fer faint­ing spells, seizures, car­diac ar­rest or sud­den death.

Be­cause peo­ple with LQTS show few signs or symp­toms, many don’t even be­come aware of their con­di­tion un­til they ex­pe­ri­ence heart fail­ure, or get an ECG for an­oth­er rea­son. The US Na­tion­al Heart, Lung, and Blood In­sti­tute es­ti­mates that one out of every 7,000 peo­ple are liv­ing with LQTS, and 3,000 to 4,000 chil­dren and young adults die of it each year.

“The con­se­quences of un­di­ag­nosed or poor­ly treat­ed Long QT Syn­drome can be de­bil­i­tat­ing and life threat­en­ing,” LQT’s sci­en­tif­ic founders An­tho­ny Rosen­zweig, David Mi­lan and Saumya Das said last year, up­on reel­ing in about $1.4 mil­lion in seed fund­ing.

Cur­rent treat­ment op­tions for LQTS are lim­it­ed to be­ta-block­ers (which come with a risk of side ef­fects like fa­tigue, dizzi­ness and short­ness of breath), and lifestyle changes such as eat­ing right and lim­it­ing ac­tiv­i­ties that can trig­ger the ab­nor­mal heart­beats. If be­ta block­ers fail, pa­tients may need surgery for an im­plantable car­diovert­er de­fib­ril­la­tor, which re­stores the heart’s nat­ur­al rhythm via elec­tri­cal shocks.

But LQT thinks it has a dif­fer­ent ap­proach. The com­pa­ny has rea­son to be­lieve that an en­zyme called Serum/Glu­co­cor­ti­coid Reg­u­lat­ed Ki­nase 1 (SGK1) plays a key role in in­ap­pro­pri­ate sodi­um reg­u­la­tion in var­i­ous ar­rhyth­mias. The idea stemmed from re­search by Mi­lan from the Leducq Foun­da­tion, Rosen­zweig and Das from Mass­a­chu­setts Gen­er­al Hos­pi­tal, and sup­port from Beth Is­rael Dea­coness Med­ical Cen­ter in Boston.

“Our re­search has un­cov­ered a unique and nov­el ther­a­peu­tic ap­proach to mod­u­late elec­tri­cal prop­er­ties of cer­tain sodi­um chan­nels which are po­ten­tial­ly im­por­tant in the treat­ment of Long QT Syn­drome,” Rosen­zweig said in a state­ment.

LQT has li­censed a slate of com­pounds from Sanofi to in­hib­it the ki­nase, the most ad­vanced of which has shown promise in the stem cells of pa­tients with LQT syn­drome, CEO Paul Truex says. The Se­ries A funds will be used to push that can­di­date in­to Phase I. And Philip Sager, a Gilead and As­traZeneca vet and in­ter­na­tion­al­ly rec­og­nized car­di­ol­o­gist, has been tapped to lead the ef­fort as CMO.

Am­pli­tude Ven­tures led the round, with a hand from Lu­mi­ra Ven­tures, Amzak Health, Alexan­dria Ven­ture In­vest­ments, found­ing in­vestor Fonds de sol­i­dar­ité FTQ, found­ing sci­en­tists and mem­bers of the man­age­ment team.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

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No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

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