Roche builds long-term case for Evrys­di with 2-year SMA da­ta; Ex­e­Vir nets $50M Se­ries A for lla­ma-de­rived Covid an­ti­body

At the two-year mark, Roche’s Evrys­di (ris­diplam) ei­ther main­tained or im­proved mo­tor func­tion from a one-year check-up in pa­tients aged 2 to 25 with Types 2 or 3 spinal mus­cu­lar at­ro­phy, the Swiss drug­mak­er said Tues­day.

Even bet­ter, Evrys­di showed no new long-term safe­ty sig­nals, and pa­tients in the sec­ond year of treat­ment ac­tu­al­ly post­ed few­er se­vere side ef­fects than in the first year, Roche said.

The SUN­FISH Part 2 study con­tin­ues an ini­tial one-year study for Types 2 and 3 SMA for Evrys­di, which snared an FDA ap­proval back in Au­gust. In ad­di­tion to stud­ies in that set­ting, Roche is look­ing at Evrys­di fol­low-ups in Type 1 SMA as well as in pa­tients who have re­ceived a pri­or SMA ther­a­py pri­or to Evrys­di and in in­fants.

“These en­cour­ag­ing re­sults con­firm that the ef­fi­ca­cy and safe­ty of Evrys­di in peo­ple with Type 2 and Type 3 SMA can be sus­tained over time,” Roche’s CMO Levi Gar­raway said in a state­ment. “There­fore, these find­ings fur­ther high­light the po­ten­tial longer-term ben­e­fit this first-of-its-kind med­i­cine can have for peo­ple of vary­ing ages and lev­els of SMA dis­ease sever­i­ty.” — Kyle Blanken­ship

Ex­e­Vir nets $50M Se­ries A for lla­ma-de­rived Covid-19 an­ti­body

What a year it’s been for lla­ma re­searchers.

The Bel­gium-based biotech Ex­e­Vir has closed a $50 mil­lion Se­ries A round to ad­vance de­vel­op­ment of its lla­ma-de­rived an­ti­bod­ies for the treat­ment and pre­ven­tion of Covid-19. Ex­e­Vir will use the fi­nanc­ing to launch a glob­al Phase Ib/II clin­i­cal tri­al of lead com­pound XVR011 that is sched­uled to start “short­ly,” the com­pa­ny said.

“In on­ly 9 months from set up, Ex­e­Vir has made fan­tas­tic progress in the de­vel­op­ment of lead com­pound XVR011 which is now ready to move in­to clin­i­cal de­vel­op­ment,” Ex­e­Vir chair­man Philippe Monteyne said in a state­ment. “We are proud that Bel­gium is at the fore­front of in­ter­na­tion­al ef­forts in cut­ting-edge re­search, in­no­va­tion and sci­ence.”

The Se­ries A comes af­ter the re­search team, in col­lab­o­ra­tion with the NIH, en­gi­neered a lla­ma an­ti­body to block the nov­el coro­n­avirus last May. Some an­i­mals, like lla­mas, pro­duce small­er an­ti­bod­ies called nanobod­ies that can po­ten­tial­ly pro­vide the foun­da­tion for in­haled Covid-19 ther­a­peu­tics.

Tues­day’s fi­nanc­ing was led by Fund+ with VIB, UCB Ven­tures, SF­PI-FPIM, V-Bio Ven­tures, and new in­vestors SRIW, Noshaq, Vives IUF, Sam­brIn­vest al­so par­tic­i­pat­ed, as well as sev­er­al Bel­gian fam­i­ly of­fices. — Max Gel­man

Pre­clin­i­cal im­muno-on­col­o­gy play­er re­verse merges with In­tec Phar­ma 

Is­rael-based In­tec Phar­ma is dip­ping its toes in im­muno-on­col­o­gy through a re­verse merg­er with the pre­clin­i­cal start­up De­coy Biosys­tems.

$NTEC shares surged 87% in pre­mar­ket trad­ing af­ter the com­pa­nies an­nounced the merg­er on Mon­day. On Tues­day morn­ing, they were down 10.9%, pric­ing around $5.31 apiece.

De­coy brings with it a “ro­bust” im­munother­a­py pipeline, CEO Jef­frey Meck­ler said in a state­ment. The com­pa­ny says it will ad­vance its plat­form to bat­tle “a va­ri­ety of tu­mor types and chron­ic vi­ral in­fec­tions.” It plans to file an IND in the sec­ond half of this year, and ini­ti­ate a Phase I tri­al in 2022 tar­get­ing sol­id tu­mors and lym­phomas.

“De­coy’s bac­te­ria-based plat­form is a nov­el modal­i­ty which has the po­ten­tial to dra­mat­i­cal­ly change how we treat can­cer and chron­ic vi­ral dis­eases,” said Roger Pomer­antz, who will be­come the com­bined com­pa­ny’s board chair.

For­mer De­coy stock­hold­ers are ex­pect­ed to own about 75% of the com­bined com­pa­ny, while In­tec share­hold­ers will take about 25%. — Nicole De­Feud­is

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.