Neil Kumar, BridgeBio

Neil Ku­mar bets near­ly $1B on what Bridge­Bio hopes will be their first big drug

Bridge­Bio CEO Neil Ku­mar fi­nal­ly won back full con­trol of the drug he let get (par­tial­ly) away.

On Mon­day, af­ter three failed at­tempts, Bridge­Bio pur­chased the last bits of stock it didn’t al­ready own in Ei­dos, buy­ing up 36.3% of one of their first sub­sidiaries for a com­bi­na­tion of cash and stock worth near­ly $1 bil­lion.

Ku­mar said in a state­ment that, with Phase III da­ta pend­ing, the buy­out would help them pre­pare a glob­al launch for what the 6-year-old com­pa­ny hopes will be their first ma­jor prod­uct, an Ei­dos rare dis­ease drug known as AG10 or aco­ramidis. But he al­so not­ed in an in­ter­view that the deal re­flect­ed where the com­pa­ny now stood: Still pri­vate in 2018, they had to take part of Ei­dos pub­lic to fund piv­otal stud­ies; but since then Bridge­Bio had raised over $700 mil­lion on pub­lic mar­kets.

“There was a time in which we couldn’t re­al­ly af­ford what we need­ed to do clin­i­cal­ly,” Ku­mar told End­points News. “Since then, Bridge­Bio’s been able to evolve as a com­pa­ny so that we have more and more re­sources, where­by we thought the re­verse would be true, and bring­ing it back in­side ful­ly would al­low the Ei­dos team to do even more.”

Al­though Bridge­Bio has yet to re­lease piv­otal da­ta for any of the nu­mer­ous drugs in its pipelines, Ku­mar has struck an up­beat note in re­cent months, say­ing they were near­ing proof for his long-run­ning strat­e­gy and lay­ing out plans to sub­mit two new drug ap­pli­ca­tions to the FDA this year. Last week, the com­pa­ny sub­mit­ted an NDA for fos­denopterin, a for­mer Alex­ion drug for an ul­tra-rare neu­ro­log­i­cal con­di­tion, de­spite the fact that they have yet to re­lease any da­ta from ei­ther of the two tri­als they say sup­ports the ap­pli­ca­tion.

(In an email, Michael Hen­der­son, CBO of Bridge­Bio and CEO of Ori­gin, the sub­sidiary de­vel­op­ing fos­denopterin, not­ed the tri­al had been restart­ed and said in­ter­im or fi­nal da­ta re­sults would be pre­sent­ed at med­ical con­fer­ences next year.)

Michael Hen­der­son

Bridge­Bio hopes fos­denopterin and a rare can­cer drug from their QED sub­sidiary will bring their first ap­provals, but Ku­mar ac­knowl­edged those drugs are not ex­pect­ed to be ma­jor com­mer­cial en­deav­ors. In­stead, he said, the big test for Bridge­Bio and Ku­mar’s the­sis that he can ac­cel­er­ate drug de­vel­op­ment by pick­ing ge­net­i­cal­ly-dri­ven ear­ly-stage as­sets and putting them un­der will lay in four dif­fer­ent da­ta read­outs.

Chief among them is the Ei­dos drug, aco­ramidis. Bridge­Bio has just fin­ished en­rolling a Phase III tri­al for transthyretin amy­loi­do­sis, a rel­a­tive­ly com­mon rare dis­ease where mis­fold­ed pro­teins se­cret­ed from the liv­er can cause po­ten­tial­ly fa­tal car­dio­vas­cu­lar events. A mod­er­ate­ly ef­fec­tive Pfiz­er drug, Vyn­daqel, for the con­di­tion, has earned $277 mil­lion through the first half of 2021 drug.

Ku­mar al­so list­ed sched­uled read­outs for an FGFR in­hibitor in a rare bone growth dis­or­der, a gene ther­a­py for an adren­al dis­or­der, and a small mol­e­cule for a cal­ci­um dis­or­der.

“Those 4 are re­al­ly what in­vestors are fo­cused on,” Ku­mar said. “If we are not suc­cess­ful on a ma­jor­i­ty of those, the the­sis that we are good as­set pick­ers and there­fore our abil­i­ty to pros­e­cute on every­thing else will be chal­lenged, it will be.”

Based on the 2018 Phase II da­ta, though, Ku­mar was con­fi­dent about the Ei­dos drug. Those re­sults showed AG10 sta­bi­lized the pro­tein in 90% of pa­tients af­ter 28 days of treat­ment. It al­so raised the con­cen­tra­tion of the pro­tein by 50% in the high dose and 36% in the low dose.

Io­n­is and Al­ny­lam have al­so de­vel­oped drugs in the field, both find­ing some ef­fi­ca­cy but lim­it­ed safe­ty. And Pfiz­er, he not­ed, was on­ly able to sta­bi­lize about 50% of the pro­tein in pa­tients.

“Think of it like a faucet,” he said. “Pfiz­er half turns off the faucet, we’re at 90%.”

He de­clined to of­fer a rev­enue pro­jec­tion, but not­ed an­a­lysts pegged the over­all space as worth be­tween $6 bil­lion and $9 bil­lion. He crit­i­cized some of the high prices that have fu­eled the mar­ket — Vyn­daquel goes for $225,000 per year — but he not­ed how large the pop­u­la­tion was for a ge­net­ic dis­ease, com­par­ing it to hy­per­trophic car­diomy­opa­thy, the dis­ease that fu­eled Bris­tol My­ers Squibb’s $13 bil­lion ac­qui­si­tion of MyoKar­dia Mon­day.

“There are very few of these dis­eases that af­fect over 100,000 pa­tients,” he said. “That por­tends rev­enues that are on the high end re­gard­less of how one ap­proach­es it.”

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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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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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

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.

A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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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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