Busy Sanofi takes a pass on one of Al­ny­lam’s rare dis­ease drugs, FDA of­fers ‘break­through’ sta­tus for PhI­II

A cou­ple of months af­ter Sanofi $SNY and Al­ny­lam $AL­NY re­tooled their RNAi col­lab­o­ra­tion so that the French phar­ma gi­ant could con­cen­trate on their he­mo­phil­ia ther­a­py while giv­ing up rights to the biotech’s patisir­an, the two part­ners are al­so go­ing their sep­a­rate ways on a late-stage drug for rare cas­es of pri­ma­ry hy­per­ox­aluria type 1.

John Maraganore

Sanofi is tak­ing a pass on an op­tion for lumasir­an (ALN-GO1), leav­ing Al­ny­lam CEO John Maraganore to take the drug in­to Phase III with a brand new break­through drug des­ig­na­tion from the FDA to speed things along.

Their drug is de­signed to “re­duce the he­pat­ic lev­els of the GO en­zyme, there­by de­plet­ing the sub­strate nec­es­sary for ox­alate pro­duc­tion,” which trig­gers se­vere ad­verse events of­ten lead­ing to kid­ney fail­ure. And Al­ny­lam is in com­pe­ti­tion with Dicer­na $DR­NA, a blood ri­val which is go­ing af­ter a dif­fer­ent tar­get in treat­ing the same dis­ease.

Sanofi may have all the R&D ac­tion it wants right now. Af­ter leav­ing the mar­ket hang­ing on promis­es of a ma­jor new deal to as­sist the strug­gling phar­ma gi­ant, Sanofi ac­quired Biover­a­tiv for $11.6 bil­lion and quick­ly fol­lowed with its ac­qui­si­tion of Abl­ynx. The Biover­a­tiv deal helped ex­plain Sanofi’s in­ter­est in gain­ing the world­wide rights to Al­ny­lam’s fi­tusir­an, giv­ing it a big­ger pipeline of he­mo­phil­ia ther­a­pies.

Yvonne Green­street

Al­ny­lam didn’t sound in any way ag­griev­ed to­day, hap­py to col­lect their third BTD from the agency and an­oth­er Phase III can­di­date as they prep what is wide­ly ex­pect­ed to be a near-term ap­proval for patisir­an, now un­der re­view.

“Based on Phase I/II clin­i­cal da­ta pre­sent­ed to date, we be­lieve lumasir­an could be a trans­for­ma­tive treat­ment for pa­tients with PH1, an ul­tra-rare dis­ease with no ap­proved ther­a­pies, in which ex­cess ox­alate pro­duc­tion leads to kid­ney fail­ure and sig­nif­i­cant mor­bid­i­ty and mor­tal­i­ty,” said Yvonne Green­street, the COO at Al­ny­lam. “We are al­so thrilled with FDA’s de­ci­sion to grant Break­through Ther­a­py Des­ig­na­tion to lumasir­an. We be­lieve this is a tes­ta­ment to the drug’s po­ten­tial to ad­dress the se­vere un­met med­ical need that PH1 rep­re­sents for pa­tients and their fam­i­lies. Al­ny­lam is now poised to ad­vance lumasir­an in­to Phase 3 in late 2018 and, as­sum­ing ap­proval, to com­mer­cial­ize lumasir­an glob­al­ly.”

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

David Meline (file photo)

Mod­er­na’s new CFO took a cut in salary to jump to the mR­NA rev­o­lu­tion­ary. But then there’s the rest of the com­pen­sa­tion pack­age

David Meline took a little off the top of his salary when he jumped from the CFO post at giant Amgen to become the numbers czar at the upstart vaccines revolutionary Moderna. But the SEC filing that goes with a major hire also illustrates how it puts him in line for a fortune — provided the biotech player makes good as a promising game changer.

To be sure, there’s nothing wrong with the base salary: $600,000. Or the up-to 50% annual cash bonus — an industry standard — that comes with it. True, the 62-year-old earned $999,000 at Amgen in 2019, but it’s the stock options that really count in the current market bliss for all things biopharma. And there Meline did well.

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Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development.

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Por­tion of Neil Wood­ford’s re­main­ing in­vest­ments, in­clud­ing Nanopore, sold off for $284 mil­lion

It’s been precisely one year and one day since Neil Woodford froze his once-vaunted fund, and while a global pandemic has recently shielded him from the torrent of headlines, the fallout continues.

Today, the California-based patent licensing firm Acacia Research acquired the fund’s shares for 19 healthcare and biotech companies for $284 million.  Those companies include shares for public and private companies and count some of Woodford’s most prominent bio-bets, such as Theravance Biopharma, Oxford Nanopore and Mereo Biopharma, according to Sky News, which first reported the sale. It won’t include shares for BenevelontAI, the machine learning biotech once valued at $2 billion.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.