Galec­to shelves high dose of IPF drug af­ter SAE im­bal­ance; Long­time Aveo part­ner hands back HER3 an­ti­body

Galec­to $GLTO is yank­ing the top dose of its IPF drug GB0139 in a clin­i­cal study af­ter the safe­ty mon­i­tor­ing board flagged an im­bal­ance of ad­verse events in the study point­ing to the ther­a­py.

Galec­to ex­pects to con­tin­ue re­cruit­ing pa­tients who are not tak­ing nintedanib or pir­fenidone at screen­ing and who would be ran­dom­ized to re­ceive GB0139 3 mg or place­bo. The DSMB rec­om­mend­ed the pa­tients ran­dom­ized to the 10mg group and all those tak­ing nintedanib or pir­fenidone should be dis­con­tin­ued from the study. Based on these rec­om­men­da­tions, the Com­pa­ny plans to work with both the study in­ves­ti­ga­tors and the ap­pro­pri­ate reg­u­la­to­ry au­thor­i­ties to im­ple­ment these changes prompt­ly.

“Based on our pri­or phase 1b/2a study of GB0139 in IPF pa­tients, we be­lieve the 3 mg dose has the po­ten­tial to be an ef­fec­tive clin­i­cal dose for these pa­tients,” said Hans Scham­bye, CEO of Galec­to. As of now, the com­pa­ny adds, they don’t ex­pect the board’s rec­om­men­da­tions to af­fect oth­er stud­ies. — John Car­roll

Aveo gets back a drug as long­time part­ner leaves the col­lab­o­ra­tion

Boston-based Aveo is get­ting back rights to a drug dubbed AV-203, an IgG1 mon­o­clon­al an­ti­body that tar­gets ErbB3 (al­so known as HER3). And the biotech says it’s glad to have con­trol again.

CAN­bridge Life Sci­ences has ter­mi­nat­ed their 5-year re­la­tion­ship, hand­ing back its rights for ex-North Amer­i­can sales. The move comes af­ter Aveo com­plet­ed a small study of the drug, see­ing a dose-lim­it­ing ad­verse event and one of two neureg­ulin pos­i­tive (NRG1+) pa­tients ex­pe­ri­enc­ing a par­tial re­sponse.

“AV-203 has demon­strat­ed ear­ly signs of ac­tiv­i­ty in an NRG1+ pa­tient that sug­gest it could have mean­ing­ful ap­pli­ca­tion in sev­er­al ar­eas of high un­met need in can­cer,” says CEO Michael Bai­ley. “We look for­ward to ad­vanc­ing AV-203 in the clin­ic as part of our strat­e­gy for de­liv­er­ing long-term val­ue from our pipeline pro­grams.” — John Car­roll

Japan­ese in­vestors back a small A round for vac­cine play­er VLP

A low-pro­file vac­cine ef­fort has raised a $16 mil­lion A round to back their de­vel­op­ment work.

The Gaithers­burg, MD-based VLP Ther­a­peu­tics has a lead pro­gram for a can­cer vac­cine, with oth­er pro­grams in place for malar­ia, dengue as well as Covid-19. The mon­ey c0mes from Miyako Cap­i­tal Co.,  So­jitz Cor­po­ra­tion, and Kon­ishiya­su Co. in Japan along with three ex­ist­ing in­vestors in the US: Robert G. Hisao­ka, SK Im­pact Fund and RJ Fund.

Masayoshi Fu­ji­mo­to, pres­i­dent and CEO of So­jitz Cor­po­ra­tion, not­ed: “We, So­jitz Cor­po­ra­tion, are very pleased to work with VLPT CEO Wataru Aka­ha­ta, an am­bi­tious sci­en­tist with con­sid­er­able ex­pe­ri­ence in vac­cine R&D, as well as with the mem­bers of the re­search team and the com­pa­ny founders who are well-versed in phar­ma­ceu­ti­cal de­vel­op­ment. We will do what­ev­er we can to help VLPT grow go­ing for­ward.” — John Car­roll

No­var­tis push­es ear­ly SMA screen­ing with more da­ta on quick in­ter­ven­tion with Zol­gens­ma

No­var­tis wants par­ents to know that their chil­dren have a lot more to gain if they’re di­ag­nosed on a pre-symp­to­matic ba­sis with SMA.

Point­ing to long-term fol­low-up da­ta from ex­ist­ing stud­ies the phar­ma gi­ant un­der­scored con­tin­ued gains for chil­dren who re­ceived the ground­break­ing gene ther­a­py be­fore they start­ed to ex­hib­it symp­toms.

“When treat­ed with Zol­gens­ma pri­or to the on­set of symp­toms, chil­dren in the SPR1NT tri­al achieved mile­stones like sit­ting, stand­ing and walk­ing at an ap­pro­pri­ate age, grew as ex­pect­ed with­out nu­tri­tion­al as­sis­tance, and re­mained free of all forms of me­chan­i­cal ven­ti­la­to­ry sup­port,” said Kevin Strauss, med­ical di­rec­tor for the Clin­ic for Spe­cial Chil­dren in Penn­syl­va­nia. “This stands in sharp con­trast to the nat­ur­al pro­gres­sion of SMA Type 1, which would oth­er­wise ren­der them help­less with­in the first year of life and un­able to swal­low, breathe, or sur­vive with­out me­chan­i­cal sup­port.” — John Car­roll

Ocuphire re­veals pos­i­tive PhI­II for eye ex­am di­lat­ing agent

Ocuphire is po­ten­tial­ly one step clos­er to see­ing a new prod­uct ap­proval fol­low­ing a pos­i­tive Phase III read­out Mon­day.

The Farm­ing­ton Hills, MI-based biotech re­leased da­ta Mon­day show­ing its Nyx­ol prod­uct for pupil di­la­tion in eye ex­ams met its pri­ma­ry end­point. In 185 in­di­vid­u­als, most­ly adults but which in­clud­ed 14 chil­dren over age 12, 49% of pa­tients re­turned to less than 0.2 mm of their base­line pupil di­am­e­ter at 90 min­utes com­pared to 7% of sub­jects treat­ed with place­bo.

That re­sult­ed in a sparkling p-val­ue of p<0.0001. Pa­tients re­ceived ei­ther Nyx­ol or place­bo, in a 1-to-1 ran­dom­iza­tion, one hour af­ter get­ting one of three di­lat­ing agents in their study eye.

“In ad­di­tion, Nyx­ol demon­strat­ed sig­nif­i­cant ben­e­fit through 6 hours across the range of com­mon­ly used my­dri­at­ic agents, light and dark iris col­ors, and age co­horts,” said Ocpuhire board mem­ber Jay Pe­pose in a state­ment.

With Mon­day’s da­ta in hand, Ocuphire is look­ing at an ear­ly 2023 NDA ap­pli­ca­tion. — Max Gel­man

BioX­cel wins BTD for de­men­tia-re­lat­ed ag­i­ta­tion pro­gram

A lit­tle over two months af­ter putting out top-line da­ta in an ear­ly stage tri­al for ag­i­ta­tion in de­men­tia and Alzheimer’s dis­ease, BioX­cel has picked up back­ing from the FDA.

Known as BX­CL501, the can­di­date re­ceived break­through ther­a­py des­ig­na­tion Mon­day for ag­i­ta­tion in de­men­tia, in­clud­ing Alzheimer’s, the com­pa­ny said Mon­day. The des­ig­na­tion was based on Jan­u­ary’s Phase Ib/II tri­al, which showed a sta­tis­ti­cal­ly sig­nif­i­cant calm­ing ef­fect com­pared to place­bo with­out se­ri­ous side ef­fects.

“Man­ag­ing de­men­tia re­lat­ed ag­i­ta­tion, specif­i­cal­ly in el­der­ly pa­tients, rep­re­sents a sig­nif­i­cant chal­lenge for physi­cians and care­givers, as there are cur­rent­ly no FDA-ap­proved ther­a­pies and off-la­bel drugs come with black box warn­ings,” CEO Vi­mal Mehta said in a state­ment.

BX­CL501 is a re­for­mu­la­tion of Pfiz­er’s 21-year-old se­da­tion drug Pre­cedex. BioX­cel cre­at­ed a dis­solv­ing film-based, sub­lin­gual ver­sion of the drug — like Lis­ter­ine strips, but placed un­der the tongue — that pa­tients ad­min­is­tered them­selves. The can­di­date pro­duces a calm­ing ef­fect with­out knock­ing a pa­tient out. — Max Gel­man

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

LLS backs 5 new can­cer drug projects with up to $50M; Trodelvy con­tin­ues to im­press with more TNBC da­ta

The Leukemia and Lymphoma Society has tapped 5 new early-stage projects to back with up to $10 million each in fresh investments. The 5 biotechs are:

— Caribou, headed by Rachel Haurwitz and co-founded by Jennifer Doudna, is working on next-gen, off-the-shelf CAR-Ts to replace the patient-derived cells now in use.

— The LLS supported NexImmune’s IPO, helping fund its work on nanoparticles that can gin up an immune response directed at cancer cells. The biotech has 2 projects now in Phase I trials.

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.