No­var­tis steps in to grab ex-US rights to pi­o­neer­ing gene ther­a­py Lux­tur­na with $170M deal

Vas Narasimhan

Just days ahead of Vas Narasimhan’s jump in­to the CEO’s spot, No­var­tis has swooped in with a $170 mil­lion deal to grab ex-US rights on Spark Ther­a­peu­tics’ Lux­tur­na, the first true gene ther­a­py to get a US ap­proval.

The phar­ma gi­ant is pay­ing $105 mil­lion in cash and putting an­oth­er $65 mil­lion on the ta­ble for near-term Eu­ro­pean reg­u­la­to­ry mile­stones. Spark $ONCE al­so qual­i­fies for a roy­al­ty stream on ex-US sales.

The deal leaves Spark on track to set up its own US mar­ket­ing op­er­a­tion for Lux­tur­na, which is de­signed for RPE65 mu­ta­tion-as­so­ci­at­ed cas­es of reti­nal dy­s­tro­phy.

No­var­tis will be spear­head­ing a com­mer­cial op­er­a­tion in Eu­rope that fol­lows the tracks of two oth­er ap­proved gene ther­a­pies which have on­ly man­aged to reach a hand­ful of pa­tients. And it faces some steep com­mer­cial bar­ri­ers for a ther­a­py that’s been priced at $850,000 — $425,000 per eye — in the US. Narasimhan, though, has put a big pre­mi­um on play­ing a lead­ing role in de­vel­op­ing and mar­ket­ing cell and gene ther­a­pies in key mar­kets.

Dan Fa­ga

The WAC price for Lux­tur­na in the US falls to­ward the high­er end of an­a­lysts’ bets, which ranged from about $600,000 to just un­der the $1 mil­lion mark for what’s in­tend­ed as a one-time treat­ment for the rare, sight-steal­ing ge­net­ic con­di­tion. Now the high­est priced ther­a­py in the coun­try — out­pac­ing drugs like Spin­raza at $750,000 for the first year of ther­a­py — it fell on Spark to come up with the right pric­ing mod­el that can per­suade pay­ers to cov­er the pro­ce­dure for a tiny group of un­der 2,000 po­ten­tial pa­tients, with few­er than 20 new pa­tients per year.

Wed­bush is skep­ti­cal that those kinds of pa­tient num­bers will play to No­var­tis’ fa­vor. They note:

No­var­tis’ re­sources and com­mer­cial ex­per­tise will not be able to over­come the main short­com­ing with Lux­tur­na pro­gram: an ad­dress­able pop­u­la­tion that is sim­ply too small to gen­er­ate mean­ing­ful val­ue.

No­var­tis will al­so face a group of sin­gle pay­er op­er­a­tions with con­sid­er­able lever­age over pric­ing, typ­i­cal­ly well un­der US rates.

“By lever­ag­ing No­var­tis’ large, ex­ist­ing com­mer­cial and med­ical in­fra­struc­ture in oph­thal­mol­o­gy, as well as its com­mit­ment to com­mer­cial­iz­ing ge­net­ic-based med­i­cines, we help en­sure that more pa­tients with con­firmed bial­lel­ic RPE65 mu­ta­tion-as­so­ci­at­ed reti­nal dy­s­tro­phy who live out­side the U.S., and im­por­tant­ly out­side of Eu­rope, have ac­cess to in­ves­ti­ga­tion­al voreti­gene nepar­vovec,” said Dan Fa­ga, chief busi­ness of­fi­cer, Spark Ther­a­peu­tics. And the mon­ey they’re get­ting will be put to­ward the R&D costs for the rest of their pipeline prod­ucts, in­clud­ing an ad­vanced pro­gram for he­mo­phil­ia B.

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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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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.

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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.

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Incyte released Phase III results from two of its clinical vitiligo programs Monday morning, saying both studies met their primary endpoints of patients achieving at least 75% improvement from baseline in repigmentation of the face. The data will likely lead Incyte to ask for approval in both the US and Europe for those older than 12 before the end of the year.

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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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Days ahead of Am­gen split, Cy­to­ki­net­ics reads out post-hoc da­ta sug­gest­ing heart drug works bet­ter in sick­er pa­tients — but can the CEO win over skep­tics?

While Cytokinetics’ heart drug technically met its primary endpoint back in November, it missed a key secondary endpoint — reduction in cardiovascular death — which eventually cost the company two partnerships. Now the team is back with data suggesting the drug works better in sicker patients, and it’s planning a trip to the FDA.

In a post-hoc analysis, which can be a very difficult sale at the FDA, Cytokinetics separated patients from the Phase III GALACTIC-HF study into four quartiles based on ejection fraction, a measurement of how well the left ventricle pumps blood with each heartbeat. Patients in the lower two quartiles — those with an EF of 22% or lower, and between 29% to 32% — saw a 15% and 17% relative risk reduction of heart failure events and cardiovascular death combined, Cytokinetics reported at ACC. No difference was seen in the upper two quartiles.

Neil Desai, Aadi Bioscience CEO (Specialised Therapeutics via YouTube)

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SPACs have become the preferred fast track for public markets over the past year, but apparently there’s still room for a good, old-fashioned reverse merger.

Cancer-focused Aadi Bioscience announced Monday that they would merge with the struggling public biotech Aerpio Pharmaceuticals. To go along with the merger, Aadi raised $155 million from private investors to commercialize their lead drug, Fyarro, which is now sitting before the FDA. Acuta Capital Partners and KVP Capital led the round.

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