Akcea, Io­n­is win over most FDA ex­perts to their rare dis­ease drug volane­sors­en, vast­ly im­prov­ing odds of suc­cess

Akcea Ther­a­peu­tics $AK­CA faced a tough up­hill bat­tle in search of an ap­proval for their rare dis­ease drug volane­sors­en to­day — and they won.

The FDA ad­vi­so­ry com­mit­tee vote on the drug — spun out from Io­n­is $IONS — was 12 for, 8 against. The FDA, of course, doesn’t have to go along. But for a rare dis­ease, they usu­al­ly do — par­tic­u­lar­ly un­der this ad­min­is­tra­tion.

Akcea’s shares shot up 20% in pre-mar­ket trad­ing on Fri­day, start­ing the day with a $1.8 bil­lion mar­ket cap, while Io­n­is saw a spike of 5%.

FDA reg­u­la­tors didn’t make the task very easy, high­light­ing their deep con­cerns with the sud­den plunge in platelets that af­flict­ed a num­ber of pa­tients through the clin­i­cal tri­als for the drug. In­ves­ti­ga­tors nev­er found a re­li­able way to screen for throm­bo­cy­tope­nia in the tri­al work, and their in­ter­nal re­view plant­ed a red flag on the fact that doc­tors pre­scrib­ing this drug would al­so like­ly be blind­sided by un­ex­pect­ed cas­es.

Io­n­is re­port­ed a lit­tle more that a year ago that it had achieved its ef­fi­ca­cy end­point in Phase III. But five pa­tients were forced out of the tri­al due to a threat­en­ing de­cline in platelet counts. Grade 4 throm­bo­cy­tope­nia oc­curred in three pa­tients, which end­ed af­ter they stopped dos­ing. There were no with­drawals due to platelet counts af­ter the com­pa­ny be­gan mon­i­tor­ing the side ef­fect.

If it does get an OK, those facts on safe­ty are like­ly to haunt the com­pa­ny’s sales ef­forts.

In its fa­vor, though, was a sol­id set of da­ta demon­strat­ing the drug’s ef­fect in reg­u­lat­ing plas­ma triglyc­eride for pa­tients with rare cas­es of fa­mil­ial chy­lomi­crone­mia syn­drome — but with­out the clear clin­i­cal im­pact that would have helped push this drug over the top with big­ger num­bers in its fa­vor.

“We thank all the mem­bers of the Com­mit­tee for their time in con­duct­ing a thor­ough and thought­ful re­view of Waylivra, a new po­ten­tial first and on­ly ther­a­py for peo­ple with fa­mil­ial chy­lomi­crone­mia syn­drome, or FCS. FCS is ul­tra-rare, se­vere and po­ten­tial­ly fa­tal with no ther­a­peu­tic op­tions. The da­ta, in­clud­ing re­sults from two phase three clin­i­cal tri­als, demon­strate clear im­prove­ment on sev­er­al im­por­tant mea­sures of dis­ease in these pa­tients. We be­lieve that these re­sults pro­vide strong sup­port to make Waylivra avail­able to treat peo­ple with FCS,” said Paula Soteropou­los, chief ex­ec­u­tive of­fi­cer of Akcea Ther­a­peu­tics. “We look for­ward to work­ing with the FDA to com­plete the fi­nal stages of reg­u­la­to­ry re­view for Waylivra. We are com­mit­ted to the FCS com­mu­ni­ty and will con­tin­ue to fo­cus on bring­ing Waylivra to peo­ple suf­fer­ing with this dev­as­tat­ing dis­ease.”

 

Exelixis CEO Michael Morrissey (file photo)

#ES­MO20: Look out Mer­ck. Bris­tol My­ers and Ex­elix­is stake out their com­bo’s claim to best-in-class sta­tus for front­line kid­ney can­cer

Now that the PD-(L)1 checkpoints are deeply entrenched in the oncology market, it’s time to welcome a wave of combination therapies — beyond chemo — looking to extend their benefit to larger numbers of patients. Bristol Myers Squibb ($BMY} and Exelixis {EXEL} are close to the front of that line.

Today at ESMO the collaborators pulled the curtain back on some stellar data for their combination of Opdivo (the PD-1) and Cabometyx (the TKI), marking a significant advance for the blockbuster Bristol Myers franchise while offering a big leg up for the team at Exelixis.

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Donald Trump and White House chief of staff Mark Meadows, before boarding Marine One (Getty Images)

Pric­ing deal col­laps­es over Big Phar­ma's re­fusal to is­sue $100 'cash card­s' be­fore the elec­tion — re­port

Late in August, as negotiations on a pricing deal with President Trump reached a boiling point, PhRMA president Stephen Ubl sent an email update to the 34 biopharma chiefs that sit on his board. He wrote that if the industry did not agree to pay for a $100 “cash card” sent to seniors before November, White House chief of staff Mark Meadows was going to tell the news media Big Pharma was refusing to “share the savings” with the elderly — and that all of the blame for failed drug pricing negotiations would lie squarely on the industry.

Dan Skovronsky, Eli Lilly CSO

UP­DAT­ED: An­a­lysts are quick to pan Eli Lil­ly's puz­zling first cut of pos­i­tive clin­i­cal da­ta for its Covid-19 an­ti­body

Eli Lilly spotlighted a success for one of 3 doses of their closely-watched Covid-19 antibody drug Wednesday morning. But analysts quickly highlighted some obvious anomalies that could come back to haunt the pharma giant as it looks for an emergency use authorization to launch marketing efforts.

The pharma giant reported that LY-CoV555, developed in collaboration with AbCellera, significantly reduced the rate of hospitalization among patients who were treated with the antibody. The drug arm of the study had a 1.7% hospitalization rate, compared to 6% in the control group, marking a 72% drop in risk.

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Seat­tle Ge­net­ic­s' Astel­las-part­nered ADC nails con­fir­ma­to­ry PhI­II in urothe­lial can­cer

Nine months after Seattle Genetics nabbed an accelerated approval for its Astellas-partnered antibody-drug conjugate Padcev, the partners said the therapy has nailed a confirmatory Phase III, proving its worth in locally advanced or metastatic urothelial cancer.

Padcev, which has widely been tapped as a potential blockbuster, scored improvements in both overall survival and progression-free survival compared to chemotherapy, causing a 30% reduction in risk of death (p = 0.001) and 39% reduction in risk of disease progression or death (p<0.00001).

#ES­MO20: Alk­er­mes of­fers their first snap­shot of a ben­e­fit for their next-gen IL-2 drug. But why did 1 pa­tient starve to death?

Everyone in the cancer R&D arena is looking to build new franchises around better drugs and combos. And one busy pocket of that space is centered entirely on creating an IL-2 drug that can be as effective as the original without the toxicity that damned it to the sidelines.

Alkermes $ALKS formally tossed its hat into the ring of contenders at virtual ESMO today, highlighting the first glimpse of efficacy for their candidate, ALKS 4230, as both a monotherapy as well as in combination with Merck’s Keytruda.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

Pfiz­er match­es Mod­er­na with their full Covid-19 tri­al blue­print — As­traZeneca says it will un­veil its pro­to­col 'short­ly'

Yesterday, after sustained public pressure as Moderna released its Phase III Covid-19 trial blueprint, Pfizer released its own full trial design for their vaccine trials. The move was designed to boost transparency and shore up public trust in the vaccines, but it also revealed differences in how the two companies are approaching the much-watched studies while failing to satisfy the demands of the fiercest advocates for transparency.

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Stronger to­geth­er? Boehringer and Mi­rati team to put first KRAS-KRAS com­bo in the clin­ic

Researchers are still waiting to see how much any of the vaunted KRAS drugs now in the clinic can, after decades of preclinical research and some early human studies, help patients. But while they do, two of the leading developers will look to see whether a KRAS-KRAS combo might pose a better shot than any KRAS alone.

Boehringer Ingelheim and Mirati have signed a collaboration to combine Mirati’s closely-watched lead KRAS inhibitor, MRTX849, in a clinical trial with the pan-KRAS blocker that Boehringer has quietly developed with high expectations behind their flashier contenders.

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#ES­MO20: Re­gen­eron, Sanofi eye an­oth­er first for their PD-1 con­tender Lib­tayo with promis­ing da­ta for on­col­o­gy niche

Regeneron and Sanofi took another step forward in the long march towards a greatly expanded market for their late-bloomer PD-1 checkpoint Libtayo.

The two occasional allies posted an objective response rate of 31% for Libtayo among 84 patients suffering from advanced cases of basal cell carcinoma at virtual ESMO. That spotlights progress for 26 patients, 5 of whom had a complete response. The data also reflect a boost in the number of responses seen from the last cut of the numbers.

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Albert Bourla, Pfizer CEO (Steven Ferdman/Getty Images)

Pfiz­er ex­ecs con­fi­dent­ly tap their top 10 block­busters-to-be. But what are the chances of sur­viv­ing PhI­II, let alone hit­ting these big peak sales es­ti­mates?

Pfizer’s top executive team doesn’t lack for confidence.

Where many Big Pharmas would be reluctant to put a peak sales figure on their late-stage drugs, Pfizer CEO Albert Bourla has shrugged off the usual diffidence to outline where the pharma giant expects to get $15 billion-plus.

The list, outlined this week during their investor presentations, is topped by 3 drugs in the $3 billion-plus peak sales category. They are:

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