Ei­dos, Avro­bio shoot for $201M as biotech IPOs con­tin­ue to roll for­ward on Nas­daq

Just a few weeks af­ter RA Cap­i­tal’s high-pro­file part­ner Ra­jeev Shah signed up to lead a $64 mil­lion round for San Fran­cis­co-based Ei­dos Ther­a­peu­tics, Bridge­Bio’s start­up fo­cused on TTR amy­loi­do­sis, the biotech is out of the IPO chute in search of $115 mil­lion or more. And they are right on the heels of an­oth­er start­up on the East Coast, Avro­bio, join­ing the lat­est wave of new ar­rivals to try their luck and sci­ence on Wall Street.

Neil Ku­mar

First, Ei­dos Ther­a­peu­tics. Bridge­Bio chief Neil Ku­mar has been bull­ish about this par­tic­u­lar sub­sidiary in the group, even though it’s up against some heavy­weight play­ers in drug de­vel­op­ment, in­clud­ing Al­ny­lam, Io­n­is and even Pfiz­er.

Their drug was ini­tial­ly ad­vanced by Is­abel­la Graef at Stan­ford and Mamoun Al­hamad­sheh, the com­pa­ny sci­en­tif­ic co-founders, who nailed down pre­clin­i­cal ev­i­dence that the drug can sta­bi­lize TTR and pre­vent the cas­cade of events that caus­es the dis­ease — a dis­ease mod­i­fy­ing ap­proach that will now head to the clin­ic. The founders say that the small mol­e­cule has a unique mode of bind­ing that “mim­ics a nat­u­ral­ly-oc­cur­ring, dis­ease-pro­tec­tive mu­ta­tion.”

In the S-1 Ei­dos takes a shot at Pfiz­er’s tafamadis, not­ing:

In our Phase 1 clin­i­cal tri­al, healthy vol­un­teers were ad­min­is­tered AG10 and at the high­est test­ed dose we ob­served greater than 95% sta­bi­liza­tion of TTR, on av­er­age, across the en­tire dos­ing in­ter­val and 100% sta­bi­liza­tion at peak blood lev­els. In con­trast, tafamidis at 20 mg and 80mg pro­vid­ed ap­prox­i­mate­ly 45% and 60% sta­bi­liza­tion at peak blood lev­els, re­spec­tive­ly, in our pre­clin­i­cal stud­ies. We be­lieve these ob­ser­va­tions of AG10’s com­par­a­tive­ly high­er sta­bi­liza­tion are at­trib­ut­able to ad­van­tages in AG10’s bind­ing mode and speci­fici­ty for bind­ing to TTR and not oth­er plas­ma pro­teins.

Ku­mar may have brought in some big play­ers on the lat­est round, but he’s kept his hands on the li­on’s share of the eq­ui­ty, with 62%, at Bridge­Bio. Shah’s group has on­ly 3.8% with Graef at 6.8%, the same as sci­en­tif­ic co-founder Mamoun Al­hamad­sheh. One of Ku­mar’s key pitch points with aca­d­e­m­ic in­ves­ti­ga­tors is that he can help them re­tain some se­ri­ous eq­ui­ty in their work, a claim which is un­der­scored by this of­fer­ing.

Next up is Avro­bio —a com­pa­ny with­out much hu­man da­ta to lean on. In fact the biotech doesn’t have much track record, pe­ri­od. But it has the back­ing of a se­ri­ous syn­di­cate, has a game plan on a gene ther­a­py strat­e­gy, and now wants to rack up around $86 mil­lion from an IPO.

Ge­off McK­ay

Op­er­at­ing in Cam­bridge, MA un­der CEO Ge­off MacK­ay, the biotech has tout­ed an ear­ly suc­cess for Fab­ry dis­ease, where their gene ther­a­py AVR-RD-01 helped spur a pa­tient’s plas­ma a-Gal A ac­tiv­i­ty in­to the nor­mal range. That helped with a $60 mil­lion crossover round for the At­las-launched biotech ear­li­er this year.

Avro­bio is long on promise and short on per­for­mance, like many of the biotechs mak­ing their way to Nas­daq this year. The com­pa­ny wants to build a mod­er­ate­ly pow­ered gene ther­a­py that can be man­u­fac­tured eco­nom­i­cal­ly and used in an out­pa­tient set­ting as they de­vel­op a slate of pro­posed once-and-done treat­ments for a set of promi­nent lyso­so­mal stor­age dis­or­ders.

And the com­pa­ny has been hit­ting the theme that the lentivi­ral ap­proach they’re us­ing has been test­ed on a num­ber of oc­ca­sions by a group of de­vel­op­ers. But they al­so not­ed that there have been some 40 ad­verse events that could have been lined to “pro­to­col treat­ment or pro­ce­dures.”

They add:

On­ly one event, the de­vel­op­ment of a left thigh mass, was a se­ri­ous ad­verse event and this mass was not at­trib­uted by the in­ves­ti­ga­tor to pro­to­col treat­ment or study pro­ce­dures.

At­las is still the biggest share­hold­er, with 26% of the eq­ui­ty.  Clarus holds 16% while SV has 12%.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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