Al­ny­lam pro­vides long-term look at Oxlu­mo, aim­ing to fur­ther carve out stake in PH1 mar­ket

Al­ny­lam is al­ready a leader in RNAi ther­a­py, hav­ing se­cured three ap­provals in the field with a fourth po­ten­tial­ly on the way. Now, the drug­mak­er is re­leas­ing new da­ta on one of its OK’ed med­i­cines that boost its long-term safe­ty pro­file.

As part of con­tin­u­ing analy­ses from its ran­dom­ized, place­bo-con­trolled Phase III tri­al for Oxlu­mo (lumasir­an), Al­ny­lam re­port­ed pos­i­tive 12-month fol­low-up da­ta in tri­al par­tic­i­pants with pri­ma­ry hy­per­ox­aluria type 1. New re­sults have shown the drug was as­so­ci­at­ed with im­prov­ing cal­ci­um de­posit lev­els in one or both kid­neys, or nephro­cal­ci­nosis, rel­a­tive to base­line.

“Nephro­cal­ci­nosis is a key in­di­ca­tor of dis­ease sever­i­ty in PH1,” said tri­al in­ves­ti­ga­tor Jef­frey Sa­land in a state­ment. “Uni­lat­er­al and bi­lat­er­al im­prove­ments in nephro­cal­ci­nosis in some pa­tients treat­ed with Oxlu­mo are a wel­come ob­ser­va­tion and are con­sis­tent with our ex­pec­ta­tion of the po­ten­tial clin­i­cal im­pact of a sus­tained and sub­stan­tial re­duc­tion in uri­nary ox­alate lev­els.”

The Cam­bridge, MA-based com­pa­ny pre­sent­ed the da­ta at the Amer­i­can So­ci­ety of Pe­di­atric Nephrol­o­gy and Pe­di­atric Aca­d­e­m­ic So­ci­eties (PAS) vir­tu­al meet­ing be­ing held over the week­end.

Mon­day’s re­sults come from 24 pa­tients who had been treat­ed with Oxlu­mo for 12 months that had valid re­nal ul­tra­sounds at base­line. Among that group, 11 of 24 pa­tients saw an im­prove­ment in their nephro­cal­ci­nosis grades rel­a­tive to base­line, four re­mained sta­ble and three had de­clined grades. The re­main­ing six didn’t have avail­able ul­tra­sounds at the fol­low-up.

More­over, 14 pa­tients with ul­tra­sounds had been di­ag­nosed with nephro­cal­ci­nosis at their base­lines. Eleven of these pa­tients showed im­prove­ment, and of that group, eight saw im­prove­ments in both kid­neys.

Al­ny­lam al­so re­port­ed that af­ter 12 months, pa­tients’ es­ti­mat­ed glomeru­lar fil­tra­tion rate (eGFR) re­mained sta­ble with lumasir­an. Kid­ney stone events, mean­while, de­creased sharply af­ter the first six months of treat­ment but then lev­eled off in the fol­low­ing six months among pa­tients in the drug arm of the tri­al.

In pa­tients who start­ed in the place­bo group but then crossed over to Oxlu­mo af­ter six months, the rate of kid­ney stones sim­i­lar­ly de­creased in the fol­low­ing half-year pe­ri­od.

All of Mon­day’s re­sults came specif­i­cal­ly from their ran­dom­ized, dou­ble-blind­ed Phase III tri­al in 39 pa­tients, age six and old­er. Al­ny­lam had al­so run an open-la­bel Phase III study in par­al­lel, but is yet to re­port fol­low-up da­ta from this study.

Oxlu­mo was ap­proved to treat PH1 back in No­vem­ber, and though the ex­act price tag is tricky be­cause it’s dosed ac­cord­ing to weight, es­ti­mates range from $380,000 to $480,000 per year. Al­ny­lam pegged peak sales at about $500 mil­lion per year, and it en­tered the mar­ket with an ini­tial show­ing of $9 mil­lion in the first quar­ter of 2021.

The re­sults come a lit­tle less than a month af­ter Dicer­na, who had pre­vi­ous­ly feud­ed with Al­ny­lam over RNAi in­tel­lec­tu­al prop­er­ty rights, sold off its Oxlu­mo roy­al­ty stake to Roy­al­ty Phar­ma for up to $240 mil­lion. Al­ny­lam and Dicer­na put aside their le­gal fight and came to­geth­er for a col­lab­o­ra­tion agree­ment in April 2020, though the lat­ter con­tin­ues work on a ri­val prod­uct.

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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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.

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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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.

In­cyte keeps rolling on top­i­cal cream for JAK in­hibitor, pass­ing two PhI­II tests in vi­tili­go

As Incyte prepares to potentially hit the market with a topical formulation of its cash cow ruxolitinib in atopic dermatitis, the Wilmington, DE-based company is beefing up its data package for another indication: vitiligo.

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.

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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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)

Patrick Soon-Sh­iong's for­mer chief sci­en­tist takes can­cer com­pa­ny pub­lic in $155M re­verse merg­er

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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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