Its sale to Al­ler­gan was called off at the last minute. One year lat­er, a biotech co-found­ed by Hen­ri Ter­meer finds a new buy­er

Lyso­so­mal Ther­a­peu­tics went in­to JP Mor­gan 2017 with a sale all but sealed. With $100 mil­lion up­front and a $48 mil­lion cash in­fu­sion to ex­e­cute on the R&D plan, Al­ler­gan lined up an ex­clu­sive op­tion to buy the Cam­bridge, MA-based biotech once the Phase I safe­ty and proof-of-con­cept da­ta for its lead drug are in.

Kees Been

The team de­liv­ered the re­sults by the end of 2018, CEO Kees Been re­called, and af­ter sev­er­al months of ne­go­ti­a­tions they had shak­en hands over a June buy­out.

“We had agreed to be ac­quired by Al­ler­gan,” he told End­points News. “And of course just a few days be­fore they were sup­posed to ac­quire us, they an­nounced their ac­qui­si­tion by Ab­b­Vie which ba­si­cal­ly ter­mi­nat­ed the deal.”

At the urg­ing of in­vestors, he spent the next year scout­ing a new buy­er for LTI’s slate of ex­per­i­men­tal Parkin­son’s dis­ease treat­ment, led by a glu­co­cere­brosi­dase en­zyme ac­ti­va­tor. The com­pa­ny fi­nal­ly set­tled on BIAL, a Por­tuguese phar­ma com­pa­ny that’s look­ing to set up a new re­search cen­ter in the US.

Been is keep­ing the up­front un­der wraps, but dis­closed that mile­stones would add up to $130 mil­lion.

All six staffers at LTI, in­clud­ing Been and CSO Pe­ter Lans­bury, have been re­tained to run the new­ly es­tab­lished BIAL Biotech.

Al­though the name has changed, the em­pha­sis re­mains on the pro­grams that LTI al­ready has go­ing, which are all dri­ven by ge­net­ic tar­gets.

The lead pro­gram, BIA 28-6156 or LTI-291, fo­cus­es on ac­ti­vat­ing one en­zyme that’s lack­ing in Parkin­son’s dis­ease pa­tients with cer­tain GBA mu­ta­tions.

“By nor­mal­iz­ing en­zyme ac­tiv­i­ty you re­store the lyso­so­mal func­tion in the cell,” he said. “With that you re­store the gly­cosph­in­golipids lipid me­tab­o­lism in the cells of the brain, which should lead to a dis­ease mod­i­fy­ing ef­fect which hope­ful­ly trans­lates in­to re­duc­ing the pro­gres­sion of the dis­ease.”

The plan for BIAL Biotech is to go straight in­to Phase II in the sec­ond half of 2021 once they nab an IND in the US (the ear­ly clin­i­cal work was done in the Nether­lands), set up reg­istries at clin­i­cal sites to iden­ti­fy the right pa­tients and com­plete long-term tox work.

One of the ways the par­ent BIAL stood out from oth­er phar­ma suit­ors, Been added, was that they agreed with this plan while oth­ers in­sist­ed on do­ing an ex­tra study with a brain imag­ing agent.

While this marks BIAL’s first of­fi­cial US foot­print, the Por­tuguese drug­mak­er al­so boasts of two out-li­censed drugs avail­able in the coun­try: Neu­ro­crine’s On­gen­tys, an add-on treat­ment to lev­odopa/car­bidopa in pa­tients with Parkin­son’s dis­ease ex­pe­ri­enc­ing “off” episodes; and the par­tial seizures drug Ap­tiom now mar­ket­ed by Sunovion.

But it’s keen to go deep­er in­to Parkin­son’s by lever­ag­ing grow­ing knowl­edge of ge­net­ic mu­ta­tions to craft a tar­get­ed ap­proach, Been said.

“They want to be­come a leader or a ma­jor play­er in the field of Parkin­son’s dis­ease fo­cused on all these sub­seg­ments of Parkin­son’s dis­ease,” he said.

That could al­so mean part­ner­ing on ge­o­gra­phies where BIAL, whose neu­ro­log­i­cal port­fo­lio is al­ready avail­able in Eu­rope, isn’t quite as ac­tive.

Co-found­ed by Hen­ri Ter­meer and Bob Car­pen­ter, LTI has ac­crued a stel­lar slate of in­vestors over the years, in­clud­ing Hat­teras Ven­ture Part­ners, At­las Ven­ture, Lil­ly Ven­tures, Sanofi-Gen­zyme BioVen­tures, Roche Ven­ture Fund and Part­ners In­no­va­tion Fund.

Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

Retrophin beefs up the rare dis­ease drug pipeline with a $517M buy­out deal

A little more than a year after Retrophin conceded the complete failure of a drug co-invented by company founder Martin Shkreli, the biotech is beefing up its rare disease pipeline through a $517 million buyout deal — fronted with $90 million in cash.

After the bell sounded Thursday, Retrophin $RTRX put out word that it’s acquiring the low-profile biotech Orphan Technologies. The buyout gives them an enzyme replacement therapy called OT-58 for the treatment of classical homocystinuria, a rare disease that is triggered by insufficient levels of an enzyme called cystathionine beta synthase.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Pfiz­er scoops up an an­tibi­ot­ic in rare M&A deal, bag­ging a vir­tu­al start­up op­er­at­ing on a shoe­string bud­get

Pfizer is stepping up with a rare antibiotics buyout deal today, grabbing Palo Alto, CA-based Arixa Pharmaceuticals in a bid to add a new oral version of avibactam, a beta lactamase inhibitor — or BLI — approved back in 2015 as part of the IV treatment Avycaz.

The Arixa acquisition follows some encouraging Phase I responses demonstrating that 60% to 80% of the oral drug is absorbed into the bloodstream. Only 7% of the IV version is absorbed orally, far below the 30% threshold Arixa has pointed to as a therapeutic threshold. The buyout gives Pfizer’s hospital group a line on a new oral combo with antibiotics like ceftibuten to go after drug-resistant cases of urinary tract infections and other ailments.

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Jean-Pierre Sommadossi, Atea president and CEO (file photo)

Roche wades deep­er in­to Covid-19 fight, ink­ing an­tivi­ral pact with $350M cash fol­low­ing Re­gen­eron deal

Roche is making its first bet on an antiviral against Covid-19 in style, shelling out $350 million in cash to grab ex-US rights.

The drug comes from Atea Pharmaceuticals, the 7-year-old biotech created by Pharmasset co-founder Jean-Pierre Sommadossi, which essentially rebranded itself as a Covid-19 fighter in May when it closed a whopping $215 million venture round. Over a dozen investors bought in, including marquee names like Bain Capital and RA Capital.