Am­gen’s star PhI­II os­teo­poro­sis drug ro­mo slammed by sur­pris­ing car­dio risk, a big plus for ri­val Ra­dius drug

Am­gen’s ro­mosozum­ab, one of its top late-stage drug prospects aimed at os­teo­poro­sis, is in some se­ri­ous trou­ble.

Late Sun­day the big biotech $AMGN and its part­ner UCB an­nounced what at first glance would ap­pear to be hap­py news: the late-stage tri­al com­par­ing ro­mosozum­ab to Fos­amax hit its pri­ma­ry and key sec­ondary end­points. But as you wind through the re­lease, some big is­sues start to rise up that raise se­ri­ous ques­tions about its fu­ture — if it has one. And that could make a big dif­fer­ence for its new­ly ap­proved ri­val at Ra­dius Health $RDUS.

Big event one: There was a no­table car­dio risk im­bal­ance be­tween ro­mo and Fos­amax; 2.5% for ro­mo and 1.9% for Fos­amax.

Big event two: The FDA wants to eval­u­ate the new set of head-to-head da­ta in siz­ing up an ap­proval. And that means no de­ci­sion is ex­pect­ed this sum­mer, as had been as­sumed ear­li­er.

Am­gen shares dropped 2.3% in pre-mar­ket trad­ing, while UCB plunged 14.5%. Ra­dius shares jumped about 15% on the news.

Umer Raf­fat at Ever­core ISI spot­ted the sig­nif­i­cance quick­ly. He not­ed:

Am­gen’s Ph 3 up­date on bone drug just now is clear­ly neg­a­tive, and very sur­pris­ing.  At this point, we are tak­ing out all ro­mo sales from the mod­el.  Con­sen­sus has ~$800M peak for this drug – if you take it out (and bake in the part­ner­ship split with UCB), EPS re­vi­sion of ~$0.32/share.  We sus­pect stock like­ly down ~3-4% on this.

That all sound­ed sus­pi­cious­ly like odanacat­ib, Mer­ck’s os­teo­poro­sis drug which the phar­ma gi­ant de­cid­ed to shelve last fall af­ter wit­ness­ing an in­crease risk of stroke for the drug. And Ge­of­frey Porges at Leerink cal­cu­lat­ed the odds of Am­gen scrap­ping the drug.

We be­lieve that the prod­uct now has on­ly a 50/50 prob­a­bil­i­ty of com­ing to mar­ket at all, and these odds are sub­ject to the re­sults of dis­cus­sions with spe­cial­ists and in­ves­ti­ga­tors, as well as full dis­clo­sure of the na­ture and sever­i­ty of the car­dio­vas­cu­lar events ob­served in both stud­ies.

Just a few weeks ago Ra­dius won their first ever drug ap­proval for abaloparatide, now be­ing lined up for the os­teo­poro­sis mar­ket as Tym­los. An­a­lysts like Ge­of­frey Porges had thought Ra­dius may try to go af­ter a unique mar­ket with a com­mer­cial strat­e­gy tak­ing in­to ac­count Eli Lil­ly’s ag­ing For­teo with the ri­val ro­mosozum­ab from Am­gen and UCB be­ing steered in­to a Ju­ly 19 PDU­FA date.

But that’s at the very least sig­nif­i­cant­ly de­layed — Ra­dius CEO Bob Ward said it nev­er was a fac­tor — as far as ro­mo is con­cerned.

Last fall an­a­lysts were talk­ing more about a straight shot at a quick ap­proval for ro­mo this sum­mer — with some sig­nif­i­cant caveats. Ro­mo — which tar­gets the scle­rostin pro­tein — fol­lowed by Am­gen’s Pro­lia (deno­sum­ab) clear­ly vault­ed the bar in Phase III for re­duc­ing ver­te­bral frac­tures, with a hefty 75% risk re­duc­tion com­pared to a place­bo plus deno­sum­ab. In­ves­ti­ga­tors al­so were able to show a bet­ter safe­ty pro­file in its pre­sen­ta­tion at the an­nu­al con­fab of the Amer­i­can So­ci­ety for Bone Min­er­al Re­search. And there was an in­crease in bone min­er­al den­si­ty among the drug arm in the study, which re­cruit­ed 7,180 high-prism post­menopausal women.

But the drug missed a key sec­ondary end­point. The drug did not sig­nif­i­cant­ly im­prove pa­tients’ risk of non-ver­te­bral frac­tures, leav­ing Ra­dius Health — which has post­ed an 86% risk re­duc­tion in ver­te­bral frac­tures — with a pos­si­ble dis­tinct ad­van­tage on that score.

Am­gen and its part­ners at UCB didn’t ig­nore the sit­u­a­tion to­day, af­ter high­light­ing the pos­i­tive.

Iris Loew-Friedrich

“We are im­pressed with the sta­tis­ti­cal­ly sig­nif­i­cant su­pe­ri­or frac­ture risk re­duc­tion of EVENI­TY over al­en­dronate, a cur­rent stan­dard of care in os­teo­poro­sis. When we think that pa­tients who have had a frac­ture are high­ly like­ly to suf­fer an­oth­er one, the im­por­tance of post-frac­ture care can­not be em­pha­sized enough,” said Iris Loew-Friedrich, UCB’s chief med­ical of­fi­cer. “We are work­ing on un­der­stand­ing the ob­served car­dio­vas­cu­lar safe­ty sig­nal and will con­tin­ue to dis­cuss these re­sults with glob­al reg­u­la­tors and ex­perts in the field.”

This is a bad twist for Am­gen. The bio­phar­ma play­er has had a se­ries of is­sues, in­clud­ing a big back­lash by pay­ers tar­get­ing its PC­SK9 drug. Watch­ing an­oth­er late-stage drug go un­der a cloud like this won’t help Am­gen’s rep with an­a­lysts.

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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Bay­er plots a ma­jor facelift at Berke­ley cam­pus, un­cork­ing a 30-year, $1.2B plan to dri­ve cell and gene ther­a­pies

Bayer first set roots in Berkeley back in 1974, when it was still operating as Miles Labs. The site has pumped out three hemophilia A treatments for distribution worldwide; but now, as the pharma continues its cell and gene therapy push, it has something bigger in mind.

Bayer is planning a 30-year revamp at the campus, which includes 918,000 square feet in new buildings and double the jobs, according to a report by the Bay Area Council Economic Institute.

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.

Steffen Schuster, ITM CEO

Ra­dio­phar­ma re­mains hot as Ger­many's ITM rais­es $109M to ad­vance neu­roen­docrine can­cer pro­gram

The world of radiopharmaceuticals has been heating up over the last few years, and Thursday saw another company focused on the field pull in a new nine-figure raise.

Germany’s ITM, or Isotopen Technologien München, scored a $109 million round of loan financing to push forward its precision oncology pipeline and fund late-stage development for its lead program. As part of the agreement, the loan will convert to shares in the event of future financial or corporate transactions, ITM said.

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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