Io­n­is rolls out promis­ing PhI­II da­ta on volane­sors­en for rare en­zyme dis­or­der, but safe­ty ques­tions linger

Louis O’Dea, Akcea

Io­n­is has nailed down pos­i­tive Phase III da­ta on its lead ex­per­i­men­tal drug volane­sors­en, used to com­bat rare cas­es of fa­mil­ial chy­lomi­crone­mia syn­drome. But the biotech $IONS is still bat­ting back ques­tions about safe­ty is­sues as it preps for fil­ings with the FDA and EMA.

In the Phase III in­ves­ti­ga­tors re­port­ed a huge drop in triglyc­erides among the 33 FCS pa­tients tak­ing the drug, hit­ting the pri­ma­ry end­point. The place­bo arm saw the lev­el of triglyc­erides go up, help­ing to il­lus­trate the treat­ment ef­fect.

The drug is de­signed to tack­le a dis­ease caused by hered­i­tary mu­ta­tions that in­hib­it the ac­tiv­i­ty of lipopro­tein li­pase, need­ed to break down triglyc­erides car­ried by chy­lomi­crons.

But re­searchers al­so not­ed that 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, but the safe­ty is­sue did not pass un­no­ticed.

“(O)ve­r­all the dis­con­tin­u­a­tion rate in the study was high­er than we had ex­pect­ed, and high­er than pre­vi­ous­ly seen in the COM­PASS safe­ty tri­al in se­vere hy­per­triglyc­eridemia (20%),” not­ed Leerink’s Paul Mat­teis.

Io­n­is’ sub­sidiary Akcea al­so tracked some ad­di­tion­al ben­e­fits, in­clud­ing the end of pan­cre­ati­tis at­tacks dur­ing the 52-week study as well as a re­duc­tion in ab­dom­i­nal pain.

Some an­a­lysts, though, will well re­mem­ber that Io­n­is stunned in­vestors last May with the news that its drug IO­N­IS-TTR­Rx trig­gered throm­bo­cy­tope­nia in pa­tients with TTR amy­loid car­diomy­opa­thy. The FDA then put the pro­gram on a clin­i­cal hold, spurring its part­ner GSK to drop plans to pur­sue a late-stage study. CEO Stan­ley Crook al­so not­ed at the time that re­searchers had seen the same is­sue in their work on volane­sors­en, spurring fears that this could be a class ef­fect.

The biotech’s shares were down 6% by ear­ly Mon­day af­ter­noon.

Since then the com­pa­ny has gone to some lengths to down­play the im­pact of throm­bo­cy­tope­nia, not­ing that physi­cians can mon­i­tor pa­tients and con­trol its im­pact.

Io­n­is has had its ups and downs along the way, but 2016 end­ed on a high note as its part­ner Bio­gen scored an FDA ap­proval for Spin­raza, which costs $750,000 for its first year.

“The suc­cess of AP­PROACH rep­re­sents an im­por­tant mile­stone to­wards our first reg­u­la­to­ry sub­mis­sions for volane­sors­en in the U.S., Eu­rope and Cana­da in 2017,” said Dr. Louis O’Dea, chief med­ical of­fi­cer, Akcea Ther­a­peu­tics.  “We seek to bring this new treat­ment as ex­pe­di­tious­ly as pos­si­ble to FCS pa­tients who have a high un­met need with po­ten­tial­ly life-threat­en­ing con­se­quences.”

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

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Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.