Dai­ichi Sankyo sinks $200M in­to new gene ther­a­py tech from Ul­tragenyx

In a leap to the gene ther­a­py space, Dai­ichi Sankyo has dropped $200 mil­lion to ac­cess Ul­tragenyx’s man­u­fac­tur­ing tech­nol­o­gy, pro­vid­ing the rare dis­ease biotech with plen­ty of cash and a stock boost amid a gen­er­al cash crunch.

For $125 mil­lion in cash and a $75 mil­lion eq­ui­ty in­vest­ment, Dai­ichi Sankyo has bought a non-ex­clu­sive li­cense to the IP around two plat­forms with which it plans to de­vel­op AAV-based gene ther­a­py prod­ucts. The Japan­ese phar­ma is pur­chas­ing the stock $RARE at $60 per share, more than a third above its cur­rent price of $44.43.

Emil Kakkis

Both de­signed to en­able mam­malian man­u­fac­tur­ing, the HeLa pro­duc­er cell line and HEK293 tran­sient trans­fec­tion plat­forms can be scal­able up to 2,000L and 200-500L, re­spec­tive­ly. In a pre­sen­ta­tion last Spring, Ul­tragenyx CEO Emil Kakkis not­ed that the for­mer is suit­ed for large in­di­ca­tions while the lat­ter has sig­nif­i­cant use in clin­i­cal tri­als and is vi­able for small­er pa­tient pop­u­la­tions.

“We are cur­rent­ly do­ing dis­cov­ery re­search for gene ther­a­py drugs us­ing AAV vec­tors as one of our fo­cused modal­i­ties to­ward sus­tained growth be­yond achieve­ment of our 2025 vi­sion,” said Masayu­ki Yabu­ta, Dai­ichi Sankyo’s head of bi­o­log­ics. “In or­der to pro­vide these drugs to pa­tients in the fu­ture, man­u­fac­tur­ing tech­nol­o­gy must be es­tab­lished ear­ly.”

An­oth­er $25 mil­lion will be due once tech trans­fer is com­plete. Dai­ichi is al­so foot­ing all costs as­so­ci­at­ed with the trans­fer.

“Though RARE and the DMTX team have like­ly been in­vest­ing in these plat­forms the last 2+ yrs, the Dai­ichi deal af­firms RARE’s 2017 pur­chase of DMTX (where the tech orig­i­nat­ed) for ~$152M as a smart move,” Jef­feries’ Mau­ry Ray­croft wrote in a note. “Re­call, this is ac­tu­al­ly the 2nd part­ner­ship around the plat­form — Bay­er part­nered w/ DMTX in 2014 for hemeA (now DTX201), which is us­ing the HeLa sys­tem w/ ini­tial clin re­sults at ASH’19 and EA­HAD’20.”

Mov­ing Out of the Clin­ic with Dig­i­tal Tools: Mo­bile Spirom­e­try Dur­ing COVID-19 & Be­yond

An important technology in assessing lung function, spirometry offers crucial data for the diagnosis and monitoring of pulmonary system diseases, as well as the ongoing measurement of treatment efficacy. But trends in the healthcare industry and new challenges introduced by the COVID-19 pandemic are causing professionals in clinical practice and research to reevaluate spirometry’s deployment methods and best practices.

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Sanofi, Glax­o­SmithK­line jump back in­to the PhI­II race for a Covid vac­cine — as the win­ners con­gre­gate be­hind the fin­ish line

Sanofi got out early in the race to develop a vaccine using more of a traditional approach, then derailed late last year as their candidate failed to work in older people. Now, after likely missing the bus for the bulk of the world’s affluent nations, they’re back from that embarrassing collapse with a second attempt using GSK’s adjuvant that may get them back on track — with a potential Q4 launch that the rest of the world will be paying close attention to.

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SCO­TUS de­clines to re­view En­brel biosim­i­lar case, tee­ing up 30+ years of ex­clu­siv­i­ty and $20B more for Am­gen’s block­buster

As the House Oversight Committee is set to grill AbbVie CEO Richard Gonzalez on Tuesday over tactics to block competition for its best-selling drug of all time, another decision on Capitol Hill on Monday opened the door for billions more in Amgen profits over the next eight years.

The Supreme Court on Monday denied Novartis subsidiary Sandoz’s petition to review a Federal Circuit’s July 2020 decision concerning its biosimilar Erelzi (etanercept-szzs), which FDA approved in 2016 as a biosimilar to Amgen’s Enbrel (etanercept). Samsung’s Enbrel biosimilar Eticovo also won approval in 2019 and remains sidelined.

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Charles Riv­er keeps adding on to its CD­MO arm, snatch­ing up a vi­ral vec­tor play­er for a tidy $350M

Contract researcher Charles River Laboratories has been on a roll recently to flesh out its manufacturing arm with a specific focus on its capabilities in gene therapy. Now, the firm is putting its name to a big check for a Maryland-based viral vector firm it thinks will add to its growing expertise in the field.

Charles River will dole out $292.5 million for gene therapy CDMO Vigene Biosciences with the possibility for an additional $57.5 million in performance-based payments, the companies said Monday. The deal will close at the start of Q3, a Charles River spokesman said.

How to man­u­fac­ture Covid-19 vac­cines with­out the help of J&J, Pfiz­er or Mod­er­na? Bi­ol­yse sees the dif­fi­cul­ties up close

When Biolyse, an Ontario-based manufacturer of sterile injectables, forged a deal with Bolivia last week to manufacture up to 50 million J&J Covid-19 vaccine doses, the agreement kicked off what will prove to be a test case for how difficult the system of compulsory licenses is to navigate.

The first problem: When Biolyse asked J&J, via a March letter, to license its Covid-19 vaccine, manufacture it in Canada and pay 5% royalties on shipments to needy, low-income countries, J&J rejected the offer, refusing to negotiate. J&J also did not respond to a request for comment.

Tim Mayleben (L) and Sheldon Koenig (Esperion)

On the heels of a sting­ing Q1 set­back, Es­pe­ri­on's long­time cham­pi­on is ex­it­ing the helm and turn­ing the wheel over to a mar­ket­ing pro

Just days after getting stung by criticism from a badly disappointed group of analysts, there’s a big change happening today at the helm of Esperion $ESPR.

Longtime CEO Tim Mayleben, who championed the company for 9 years from early clinical through a lengthy late-stage drive to successfully get their cholesterol drug approved for a significant niche of patients in the US, is out of the C suite, effective immediately. Sheldon Koenig — hired at the end of 2020 with a resume replete with Big Pharma CV sales experience —  is stepping into his place, promising to right a badly listing commercial ship that’s been battered by market forces.

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No­var­tis' En­tresto takes its 2nd fail­ure of the week­end at ACC, show­ing no ben­e­fit in most dire heart fail­ure pa­tients

Novartis’ Entresto started the ACC weekend off rough with a trial flop in heart attack patients, slowing the drug’s push into earlier patients. Now, an NIH-sponsored study is casting doubt on Entresto’s use in the most severe heart failure patients, another black mark on the increasingly controversial drug’s record.

Entresto, a combination of sacubitril and valsartan, could not beat out valsartan alone in an outcomes head-to-head for severe heart failure patients with a reduced ejection fraction (HFrEF), according to data presented Monday at the virtual American College of Cardiology meeting.

Matt Gline (L) and Vivek Ramaswamy

In­sid­er ac­count of Roivan­t's SPAC deal — and that $7.3B val­u­a­tion — re­veals a few se­crets as Matt Gline po­si­tions the com­pa­ny as the new ‘Big Phar­ma’

It was Oct. 7, 2020, and Matt Gline wasn’t wasting any time.

The CEO of Roivant had word that KKR vet Jim Momtazee’s SPAC had priced late the night before, triggering a green light for anyone interested in pursuing a big check for future operations and riding the financial instrument to Nasdaq. So he wrote a quick email congratulating Momtazee, whom he knew, for the launch.

Oh, and maybe Momtazee would like to schedule something with Gline and his executive chairman, Roivant founder Vivek Ramaswamy, to chat about Roivant and its business?

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Josep Bassaganya-Riera, Landos Biopharma CEO (Landos)

Per­cep­tive's Chi­na up­start Lian­Bio con­tin­ues swing­ing deals, team­ing up with lead Xon­toge­ny biotech Lan­dos in IBD

One of China’s biggest up-and-comers has a brand new partner, and it’s one whose backers are likely familiar with the other’s.

Perceptive’s LianBio has secured a collaboration with Landos Biopharma, the lead company in Chris Garabedian’s Xontogeny fund, to develop and market two programs in Greater China and other countries in the region, the biotechs announced Monday morning. In exchange, Landos is getting an upfront payment of $18 million, up to $200 million in milestones and royalties on sales in the licensed territories.