Af­ter putting aside a bit­ter le­gal feud, Al­ny­lam and Dicer­na chiefs make nice with an RNAi col­lab­o­ra­tion

John Maraganore and Dou­glas Fam­brough used to be at each oth­er’s throats as Al­ny­lam pur­sued claims that its RNAi ri­vals at Dicer­na had im­prop­er­ly pur­loined the IP it had picked up from Mer­ck in a bar­gain base­ment fire sale.

Dou­glas Fam­brough

But that was all set­tled up close to 2 years ago with a set­tle­ment from Dicer­na’s Fam­brough. And now the two are mov­ing ahead in a close R&D part­ner­ship that makes them col­lab­o­ra­tors on a cou­ple of key dis­ease tar­gets.

In the pact an­nounced this morn­ing, the 2 new al­lies will pool their re­spec­tive RNAi drugs for al­pha-1 liv­er dis­ease, let­ting Dicer­na move ahead on the de­vel­op­ment front to see whether 1, or both, should be ad­vanced through a piv­otal pro­gram. Dicer­na will foot the bill on that ef­fort while Al­ny­lam gains ex-US com­mer­cial rights to what­ev­er comes out the reg­u­la­to­ry side with a win.

Both of these drugs — ALN-AAT02 and DCR-A1AT — are in Phase I/II at the mo­ment.

That puts them be­hind Ar­row­head, which is in a Phase II/III piv­otal with their ri­val and start­ed dos­ing pa­tients in a small mid-stage study late last year fo­cused on pa­tient re­spons­es.

John Maraganore

Then Al­ny­lam and Dicer­na are cross-li­cens­ing drugs for pri­ma­ry hy­per­ox­aluria. That cov­ers Al­ny­lam’s lumasir­an tar­get­ing gly­co­late ox­i­dase for the treat­ment of PH type 1 and Dicer­na’s ne­dosir­an tar­get­ing lac­tate de­hy­dro­ge­nase A for the treat­ment of PH types 1, 2, and 3.

They each are of­fer­ing the oth­er a roy­al­ty stream on glob­al sales.

The al­liance shoves the old dis­pute in­to the his­to­ry books, rel­e­gat­ed now to a chap­ter on IP law­suits. Al­ny­lam had been up­set af­ter watch­ing Mer­ck staffers al­leged­ly make off with the IP they were buy­ing from the phar­ma gi­ant, on­ly to wind up at their ri­val. Dicer­na’s counter-claims spot­light­ed some com­pelling anec­dotes about how eas­i­ly re­search leaked out of Mer­ck af­ter the com­pa­ny de­cid­ed to punt their R&D flop.

Dicer­na claimed that Mer­ck ex­ec­u­tives prac­ti­cal­ly did every­thing but help car­ry suit­cas­es loaded with re­search out the door as they helped line up new jobs for the de­part­ing staffers. And their case de­tailed nu­mer­ous in­stances of just how help­ful they could be.

Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,600+ biopharma pros reading Endpoints daily — and it's free.

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,600+ biopharma pros reading Endpoints daily — and it's free.

Cedric Francois, Apellis CEO (Optum via YouTube)

UP­DAT­ED: So­bi bets $250M cash, about $1B in mile­stones for rights to a C3 ther­a­py be­ing pushed through 5 piv­otal tri­als

A couple years after licensing Novimmune’s emapalumab and turning around a quick FDA OK, Stockholm-based Sobi is betting up to $1.2 billion for rights to another rare disease drug.

The company is shelling out $250 million upfront and adding up to $915 million in milestones for rights to develop and commercialize Apellis Pharmaceuticals’ drug pegcetacoplan outside the US. Together, the companies will see the systemic C3 therapy through five registrational trials in hematology, nephrology and neurology.

Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,600+ biopharma pros reading Endpoints daily — and it's free.

Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,600+ biopharma pros reading Endpoints daily — and it's free.

Yung Chyung, Scholar Rock CMO (Business Wire)

A dark horse en­trant in­to the spinal mus­cu­lar at­ro­phy field dou­bles its val­ue on some PhII da­ta

The last four years have seen a sudden explosion in treatments for spinal muscular atrophy, a neurodegenerative condition that once led patients — often young ones — with a grim prognosis and no options. The prognosis still isn’t rosy, but now there are three FDA-approved options, enough to make the choice of one difficult.

Now a fourth potential option has entered the mix. Today, Scholar Rock announced the results from a proof-of-concept testing their SMA drug by itself and in combination with Ionis’ Spinraza, showing that all patient cohorts improved on standard scales used for measuring motor function in people with SMA.

Charles Baum, Mirati CEO

UP­DAT­ED: 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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,600+ biopharma pros reading Endpoints daily — and it's free.

Kevin Conroy (Exact Sciences)

UP­DAT­ED: A month af­ter Il­lu­mi­na's big Grail buy­out, Ex­act Sci­ences scoops up liq­uid biop­sy ri­val Thrive for a rel­a­tive bar­gain

Illumina is going to have a lot of work to do to prove Grail was worth those $8 billion.

Today, Exact Sciences announced that it will acquire Thrive, Grail’s chief rival among the early cancer detection startups, for a sizeable but relatively moderate $2.15 billion. The yawning gap in part reflects the vast differences in capital that have been invested to date in each company.  But both have gone toe-to-toe over the last year and a half, with Grail having published data in over 50 cancers but Thrive recently beating them to a key test for liquid biopsy companies.

Three years in­to a PhI­II pro­gram for a failed Duchenne MD drug, Cataba­sis hauls down the flag and ad­mits de­feat

Three years ago, Catabasis CEO Jill Milne and the crew insisted they had found good reason for great cheer once they plumbed the data from their failed study for the Duchenne MD drug edasalonexent. Plunging into the extended open-label data, they said, you could find solid evidence of efficacy. And that justified a try in Phase III.

But they were wrong.

Monday, after the bell, the little biotech acknowledged that their pivotal attempt following the mid-stage flop was another failure. The primary, change in baseline on the North Star Ambulatory Assessment, and the secondary on timed function tests both came up short of statistical significance.