Arde­lyx scraps PhI­II drug af­ter re­searchers raise a safe­ty is­sue for hy­per­kalemia pa­tients

The roller coast­er ride at Arde­lyx is con­tin­u­ing to­day with news that one of their late-stage as­sets is be­ing scrapped.

With their lead drug tena­panor be­ing slow­ly guid­ed to reg­u­la­tors and a hoped-for mar­ket show­down for ir­ri­ta­ble bow­el syn­drome, Arde­lyx $ARDX re­port­ed that it has de­cid­ed to scut­tle its Phase III pro­gram for RDX7675 in pa­tients with hy­per­kalemia af­ter track­ing a safe­ty is­sue — drug-re­lat­ed re­duc­tion in serum bi­car­bon­ate, which can trig­ger a de­ci­sion to stop treat­ment — which es­sen­tial­ly blew up its plans to mar­ket this treat­ment for chron­ic cas­es.

The set­back, though, didn’t seem to faze in­vestors. Arde­lyx shares, which have fol­lowed a neck-snap­ping pace of ups and downs, are up a bit this morn­ing. And Leerink’s Sea­mus Fer­nan­dez tells us why:

“(O)ur re­cent ini­ti­a­tion and en­thu­si­asm for ARDX is geared al­most en­tire­ly to­ward the oppt’y for tena­panor in hy­per­phos­phatemia. Mgmt ex­pects the ter­mi­na­tion of ‘7675 to re­sult in cash sav­ings of ~$40M and be­lieves the com­pa­ny now has cash run­way in­to 2019.”

Mike Raab

“The goal for RDX7675 was to de­vel­op a palat­able prod­uct that could be tak­en chron­i­cal­ly to ad­dress an im­por­tant med­ical need for pa­tients with hy­per­kalemia (high blood lev­els of potas­si­um). We are pleased by the ac­tiv­i­ty ob­served; how­ev­er, the unan­tic­i­pat­ed bi­car­bon­ate side ef­fect cre­ates a bar­ri­er for RDX7675, which we be­lieve could lim­it its chron­ic use,” said Mike Raab, chief ex­ec­u­tive of­fi­cer of Fre­mont, CA-based Arde­lyx, in a state­ment.

The biotech has plen­ty left to do. There’s an ear­ly-stage ther­a­py for hy­per­kalemia called RDX013 that will be moved up. And the next Phase III pro­gram for tena­panor for hy­per­phos­phatemia in end-stage re­nal dis­ease pa­tients on dial­y­sis is be­ing en­rolled, with a con­trol arm be­ing added af­ter reg­u­la­tors told them they need­ed more safe­ty da­ta. An ap­pli­ca­tion for tena­panor as a new treat­ment for IBS-C is al­so in the works.

When Arde­lyx ex­ecs rolled out their first round of Phase III da­ta on their IBS drug tena­panor, they were con­fi­dent that they were play­ing a win­ning hand, with sta­tis­ti­cal­ly sig­nif­i­cant re­sults in beat­ing out a place­bo for eas­ing ab­dom­i­nal pain and in­creas­ing bow­el move­ments.

In­stead they were crushed af­ter some an­a­lysts could bare­ly sti­fle a yawn over per­fect­ly ap­prov­able da­ta that didn’t mea­sure up so well against new da­ta from a ri­val drug from Syn­er­gy $SGYP called Tru­lance as well as Iron­wood’s fran­chise $IR­WD drug Linzess. Then, a few weeks ago, the stock rock­et­ed up on bet­ter com­par­i­son da­ta.

There’s nev­er a dull mo­ment at Arde­lyx.

Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

Left to right, top to bottom: Carl Gordon, Adam Stone, Peter Moglia, David Schenkein, Robert Nelsen, Carol Gallagher; Srinivas Akkaraju, Ray Debbane, Jim Flynn, Peter Kolchinsky, Thilo Schroeder, Brad Bolzon

The top 100 bio­phar­ma ven­ture in­vestors at the mega­bil­lions deal ta­ble

The VC crowd took a step back last year, but nevertheless maintained a furious pace of new investments in therapeutic tech platforms and biotech startups. And the top 100 players completely dominated the megabillions game.

Just looking at the number of deals done by each of the top 100, OrbiMed came in at the top, with 20, followed by Alexandria (18), Perceptive (16) and the ubiquitous RA Capital at 16. It’s impossible to say exactly how much they invested in total — those numbers are only rarely provided — but it is clear from the numbers assembled by Chris Dokomajilar at DealForma who’s most likely to be found sitting at the table during the go-go days of biotech investing.

Dokomajilar tracked $14.06 billion in biotech venture investing last year, a dip from the frenzied pace of $16.02 billion in 2018 and more than $10 billion higher than he recorded for 2010, as the economy was recovering from a profound economic crisis.

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Lars Fruergaard Jørgensen, chief executive officer of Novo Nordisk A/S, (via Getty Images)

The list of the 11 block­busters-to-be in line for a 2020 launch high­light agony and ec­sta­sy of drug R&D

For all the talk about unmet medical need and patients first and so on, the key criteria investors watch for any new drug in the pipelines is peak sales projection. Are you going to hit the blockbuster mark, at $1 billion-plus, or are you going to be an also-ran in the sales department?

Of course, analysts’ peak sales projections by themselves are of limited value in many cases. When the PCSK9 drugs started arriving 5 years ago, Repatha was billed as a $2.5 billion peak earner. They’re nowhere near that, with new competition threatening current levels. And if Biogen’s controversial Alzheimer’s drug aducanumab (submission planned but not on the list) is approved, per chance, will payers cover it?

Maybe not. And then those $10 billion in peak sales assumptions would go straight down the drain.

But, analysts are analysts, and peak sales projections have to be factored in when assessing the top experimental drugs up for a launch in the year ahead.

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For­mer No­var­tis ex­ec pleads guilty to gener­ic price fix­ing

On Friday, Hector Armando Kellum — a former senior executive at Novartis’ Sandoz unit plead guilty to charges of conspiring to fix the prices of products, including eczema and psoriasis treatment clobetasol and anti-fungal therapy nystatin triamcinolone, the Justice Department said.

His alleged partners-in-crime included Ara Aprahamian, a former sales and marketing executive at Taro Pharmaceutical Industries, who was also indicted in Philadelphia earlier this month for price-fixing and bid-rigging.

AstraZeneca executives at earnings conference, February 2020 (Natalie Grover for Endpoints News)

Coro­n­avirus out­break clouds As­traZeneca's Q4 re­sults and 2020 out­look

LONDON — With nearly a fifth of total sales coming from China, AstraZeneca counts the coronavirus-hit nation as one of its biggest growth drivers. The impact of the deadly epidemic has unsurprisingly weighed on the British drugmaker’s fourth-quarter results.

On Friday, AstraZeneca posted fourth-quarter sales of $6.25 billion, just shy of Wall Street estimates. Nearly $1.2 billion of that product revenue came from China.

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Carl June, via Getty

Carl June on CRISPR, CAR–T and how the Viet­nam War dropped him in­to med­i­cine

In August of 2011, Carl June and his team published a landmark paper showing their CAR–T treatment had cleared a patient of cancer. A year-to-the-month later, Jennifer Doudna made an even bigger splash when she published the first major CRISPR paper, setting off a decade of intense research and sometimes even more intense public debate over the ethics of what the gene-editing tool could do.

Last week, June, whose CAR–T work was eventually developed by Novartis into Kymriah, published in Science the first US paper showing how the two could be brought together. It was not only one of the first time scientists have combined the groundbreaking tools, but the first peer-reviewed American paper showing how CRISPR could be used in patients.

New Sanofi chief Paul Hud­son takes the ax to his ex­ec­u­tive com­mit­tee, chop­ping 4 key play­ers

Sanofi’s new CEO is jettisoning 4 top execs from the pharma giant’s executive committee as he reorganizes the company and attempts a reboot of the global organization. And each of them were promoted to their current positions over the last 2 or 3 years, as ex-CEO Olivier Brandicourt tried to put his own stamp on the company.

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Palleon's first CSO ex­plains how they (and she) got here; En­ter­prise taps Roche and No­var­tis vet David Mor­ris as CMO

For a decade, Li Peng built antibodies at the MedImmune labs outside DC: A bispecific Amgen is now testing in GI cancer, an Il-5 for certain kinds of asthma, another that impacts natural killer cells near tumors.

It was a sensible approach. The first antibody to treat cancer was introduced not long before she began grad school and the hunt has played a major role in biotech for much of Peng’s career.

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New en­tries, price hikes spur changes among the 20 most ex­pen­sive drugs — though not the kind politi­cians like to see

No matter how hard politicians in Washington DC pound on pharma companies for setting astronomical drug prices, they can’t seem to change one thing: The most expensive keep getting more expensive.

List prices for the most costly therapies, of course, is not fully representative of all prescription drugs for a variety of reasons, not the least because patients almost never end up paying that full amount — a fact the biopharma world likes to highlight. But the update on GoodRx’s running list of the 20 most expensive drugs in the US is still illustrative of the unabating trend of megapricing.

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