The Med­i­cines Com­pa­ny touts fresh ev­i­dence that their PC­SK9si can durably bat down LDL

John Maraganore, Al­ny­lam

The ex­ec­u­tive crew at The Med­i­cines Com­pa­ny say that they re­main on track with an ear­ly-stage PC­SK9 that re­li­ably knocked down lev­els of bad cho­les­terol, clear­ing one more hur­dle in a long jour­ney aimed at de­liv­er­ing a new ther­a­py that can beat out the pi­o­neers in the field with a drug that will on­ly need to be ad­min­is­tered two, three or four times a year.

There’s noth­ing new about PC­SK9 drugs, de­signed to slash LDL. Am­gen and the Re­gen­eron/Sanofi team de­liv­ered back-to-back ther­a­pies that do the same thing. But The Med­i­cines Com­pa­ny be­lieves it can roll up the mar­ket with a new and bet­ter ap­proach in-li­censed from Al­ny­lam that will be much eas­i­er to re­main com­pli­ant on.

Clive Mean­well, The Med­i­cines Com­pa­ny

The top-line in­ter­im analy­sis show­ing that PC­SK9si was con­tin­u­ing to look ef­fec­tive at the 90-day mar­ket — along with an ab­sence of safe­ty is­sues — stoked fresh hopes that the biotech com­pa­ny could be on­to some­thing. And the com­pa­ny is promis­ing an even more ad­vanced peak at the da­ta at the big AHA meet­ing next month.

“Mgmt cites the da­ta as “ro­bust” and that the LDL curves are ‘tight,'” telegraphed Ever­core ISI’s Umer Raf­fat Tues­day morn­ing.  “Com­pa­ny plans on head­ing in­to an ag­gres­sive Ph 3.”

That was en­cour­ag­ing enough for in­vestors to dri­ve shares of The Med­i­cines Com­pa­ny $MD­CO up 6% this morn­ing.

Last year, The Med­i­cines Com­pa­ny CEO Clive Mean­well and Al­ny­lam CEO John Maraganore out­lined plans for a years-long ef­fort aimed at dis­rupt­ing what is wide­ly ex­pect­ed to be­come a large mar­ket for these PC­SK9 cho­les­terol drugs. And Maraganore has told End­points that he be­lieves their drug is a sure-fire block­buster in the mak­ing.

Med­i­cines Com­pa­ny CEO Clive Mean­well had this to say to­day:

“These com­pelling in­ter­im da­ta af­firm PC­SK9si’s high­ly-com­pet­i­tive pro­file and val­i­date PC­SK9si’s game-chang­ing po­ten­tial. We look for­ward to pre­sent­ing re­sults from the ORI­ON-1 study, in­clud­ing Day 180 fol­low-up for up to 200 pa­tients, at the AHA meet­ing on No­vem­ber 15, 2016.”

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 plan to near­ly dou­ble 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.

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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David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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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. 

Jason Kelly. Mike Blake/Reuters via Adobe

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.