Acor­da writes off an­oth­er R&D pro­gram as Ampyra flops in stroke study

Ron Co­hen

Six months af­ter Acor­da was forced to write off one of its drug can­di­dates in the wake of a tri­al flop, the biotech is back with more bad news for in­vestors. Its fran­chise drug Ampyra (dal­fam­pri­dine) failed a piv­otal study for help­ing stroke vic­tims walk bet­ter. And Acor­da is writ­ing the ef­fort off as a los­er.

A lit­tle more than three years ago Acor­da $ACOR was stoked by ev­i­dence of suc­cess in a small study of the same pa­tient pop­u­la­tion. But an analy­sis of the da­ta this time showed that the drug es­sen­tial­ly lined up with a place­bo when put to a ma­jor test.

Acor­da’s shares were dent­ed again by the news, drop­ping 13% in pre-mar­ket trad­ing. Their shares have lost half their val­ue over the course of the past 12 months.

Eval­u­at­ing da­ta from 368 pa­tients in the study, 23 of 121 pa­tients tak­ing 10 mg dal­fam­pri­dine, 17 of 121 pa­tients tak­ing 7.5 mg of the drug and 17 of 126 tak­ing a place­bo demon­strat­ed at least a 20% im­prove­ment in a two-minute walk test. That may have re­flect­ed a slight drug ef­fect, but it wasn’t near­ly good enough for an ex­pand­ed in­di­ca­tion.

Back in May Acor­da was al­so forced to fi­nal­ly punt the epilep­sy pro­gram for Plumi­az, re­ject­ed in 2013, af­ter the nasal spray for­mu­la­tion for di­azepam failed a bioe­quiv­a­lence study. Plumi­az was one of the most ad­vanced R&D ef­forts at Acor­da. And while it was con­sid­ered less po­ten­tial­ly lu­cra­tive than the in­haled lev­odopa ther­a­py CVT-301 ($500 mil­lion peak sales es­ti­mate by the com­pa­ny) and tozadenant ($400 mil­lion peak), the biotech had made it a key part of its $1 bil­lion-plus plan to boost rev­enue by a block­buster mea­sure of suc­cess.

Acor­da CEO Ron Co­hen, BIO’s chair, ac­knowl­edged the most re­cent fail­ure and then used it as an ex­am­ple of the high-risk na­ture of drug de­vel­op­ment — a com­mon theme of his as the in­dus­try or­ga­ni­za­tion looks to steer the cur­rent de­bate over drug pric­ing to­ward fa­vor­able treat­ment for biotech in­no­va­tion.

“We are dis­ap­point­ed by this out­come. The study in­di­cat­ed there was ac­tiv­i­ty re­lat­ed to walk­ing in peo­ple with PSWD, as sug­gest­ed by the pri­or Phase 2 study, but over­all this was not suf­fi­cient­ly clin­i­cal­ly mean­ing­ful. I want to ex­press our grat­i­tude to the study par­tic­i­pants, their care part­ners and clin­i­cians, who gave their time and com­mit­ment to this re­search. This out­come un­der­scores the risks that com­pa­nies in the bio­phar­ma­ceu­ti­cal in­dus­try must take in or­der to de­vel­op in­no­v­a­tive med­i­cines. Over the past three years, we have suc­cess­ful­ly di­ver­si­fied our pipeline port­fo­lio to ac­count for this risk. We plan to fo­cus R&D re­sources on de­vel­op­ing our promis­ing late-stage Parkin­son’s dis­ease ther­a­pies, CVT-301 and tozadenant, as well as ad­vanc­ing our ear­li­er stage as­sets, CVT-427 in mi­graine, SYN120 in Parkin­son’s dis­ease de­men­tia, and rHIgM22 in MS.”

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.