Lon­don biotech com­pletes $2.5M mi­cro IPO; AGTC touts ear­ly gene ther­a­py da­ta — but in­vestors keep their dis­tance

These days, you make do with what you can get from the pub­lic biotech mar­kets.

For its first flight across the pond, Lon­don-based OKYO Phar­ma raised $2.5 mil­lion from its Nas­daq IPO, pric­ing 625,000 shares at $4 a pop.

A pre­clin­i­cal play­er, the biotech is work­ing on new treat­ments for in­flam­ma­to­ry dry eye dis­eases and oc­u­lar pain.

AGTC touts ear­ly gene ther­a­py da­ta — but in­vestors are keep­ing their dis­tance

Af­ter years of dis­ap­point­ing set­backs, Ap­plied Ge­net­ic Tech­nolo­gies Corp says it fi­nal­ly has some good news to re­port — even if in­vestors are still in wait-and-see mode.

The small biotech, whose shares have tum­bled in­to pen­ny stock ter­ri­to­ry, has post­ed what it called “com­pelling” re­sults at a three-month in­ter­im analy­sis for a Phase II tri­al of AGTC-501, its gene ther­a­py for X-linked re­tini­tis pig­men­tosa.

Al­though the tri­al’s main goal was to test the ther­a­py’s safe­ty and tol­er­a­bil­i­ty — and it looked clean on that front — AGCT said among the group who got the high­er dose, 62.5% qual­i­fied as re­spon­ders on the pri­ma­ry ef­fi­ca­cy end­point of im­prove­ments in vi­su­al sen­si­tiv­i­ty. A to­tal of 13 male pa­tients were split in­to two groups and treat­ed with two dif­fer­ent dos­es; the re­spon­der rate dropped to 25% in the low-dose arm.

“We be­lieve that if we achieve sim­i­lar re­sults in the Vista Phase 2/3 clin­i­cal tri­al, we will have a broad and com­pelling body of da­ta to sup­port the sub­mis­sion of a BLA to the FDA, and to en­able a dif­fer­en­ti­at­ed and high­ly com­pet­i­tive prod­uct pro­file.”

But the in­vestor re­ac­tion was “some­what mut­ed,” ob­served Stifel an­a­lyst Dae Gon Ha.

“(T)he stock be­ing flat af­ter hours, we think, re­flects in­vestor in­cli­na­tion to await ad­di­tion­al gran­u­lar­i­ty on to­mor­row’s CC and as­sur­ances that SKY­LINE — and the sub­se­quent Ph.2/3 VISTA tri­al — can be per­ceived as de-risked, com­fort­ably plac­ing AGTC as a lead­ing pi­o­neer in this space,” he wrote in a note.

Found­ed in 1999, Flori­da-based AGTC once gar­nered the fa­vor of Bio­gen but has since bro­ken off its part­ner­ship and strug­gled af­ter flag­ging safe­ty con­cerns in an­oth­er tri­al.

Ul­tragenyx beefs up gene ther­a­py pipeline 

As rare dis­ease spe­cial­ist Ul­tragenyx con­tin­ues to bag new pro­grams for its gene ther­a­py port­fo­lio, the lat­est ad­di­tion should fit right in.

The com­pa­ny is buy­ing a gene ther­a­py can­di­date for San­fil­ip­po syn­drome type A from Abeona. In ex­change for an undis­closed amount, Abeona will re­ceive tiered roy­al­ties and com­mer­cial mile­stone pay­ments on ABO-102.

San­fil­ip­po syn­drome type A is al­so known as mu­copolysac­cha­ri­do­sis type III (MPS III), with MPS be­ing a group of meta­bol­ic dis­or­ders caused by miss­ing or dys­func­tion­al lyso­so­mal en­zymes. One of Ul­tragenyx’s mar­ket­ed drugs, Mep­se­vii, treats MPS VII.

“Our team’s ex­per­tise in MPS and gene ther­a­py clin­i­cal de­vel­op­ment makes this pro­gram a seam­less in­te­gra­tion, and it has the po­ten­tial to be our first gene ther­a­py to mar­ket,” said Ul­tragenyx CEO Emil Kakkis.

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.

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.

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.

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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Mi­to­chon­dria biotech re­ceives Morn­ing­side buy­out of­fer af­ter PhII fail; On­col­o­gy mi­cro­cap with­draws IPO plans

Not even two months after reporting a Phase II fail for its lead drug candidate, Stealth BioTherapeutics is now being eyed for a potential acquisition, with one firm wanting to take it private.

Stealth reported Monday that Morningside Venture Investments sent Stealth’s board of directors a non-binding preliminary proposal to acquire all outstanding shares not yet owned by Morningside for 2.6 cents per share and 31.3 cents per ADS.