Find­ing a win­ner in VX-445, Ver­tex hus­tles CF com­bo to reg­u­la­tors on both sides of At­lantic

The ver­dict is in from Ver­tex’s clin­i­cal team.

The biotech was pre­sent­ed with a pleas­ant dilem­ma in March when a cock­tail for cys­tic fi­bro­sis mix­ing VX-​445, teza­caftor and iva­caftor passed Phase III with fly­ing col­ors, match­ing the re­sults they got for VX-659 with the oth­er two drugs, al­so known as Symdeko and Ka­ly­de­co. That left ob­servers hang­ing as to which com­bo will head to reg­u­la­tors.

Ver­tex ul­ti­mate­ly de­cid­ed to go with VX-445 — af­ter re­view­ing some more da­ta it’s dis­clos­ing to­day.

Resh­ma Ke­wal­ra­mani Ver­tex

At week 24 of treat­ment, pa­tients with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion on the drug main­tained ab­solute im­prove­ment in per­cent pre­dict­ed forced ex­pi­ra­to­ry vol­ume in one sec­ond of 14.3% (p<0.0001). For a key sec­ondary end­point, the same group of pa­tients saw a 63% re­duc­tion in the an­nu­al­ized rate of pul­monary ex­ac­er­ba­tions com­pared to place­bo, with the same stel­lar p-val­ue.

As pre­vi­ous­ly re­port­ed, pa­tients on the VX-445 triple had their ppFEV1 im­prove by an av­er­age of 13.8% at week 4. In a sep­a­rate tri­al for VX-​659, the num­ber was 14%.

“Peo­ple with CF who have one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion are the largest re­main­ing group of CF pa­tients with­out a treat­ment op­tion for the un­der­ly­ing cause of their dis­ease,” CMO Resh­ma Ke­wal­ra­mani said in a state­ment.

The biotech fur­ther not­ed that the de­ci­sion “was based on a de­tailed as­sess­ment of mul­ti­ple fac­tors, in­clud­ing fa­vor­able pro­files for safe­ty, tol­er­a­bil­i­ty and drug-drug in­ter­ac­tions, the abil­i­ty for co-ad­min­is­tra­tion with hor­mon­al con­tra­cep­tives, and the lack of pho­to­sen­si­tiv­i­ty.”

Ver­tex will now take these da­ta, along­side an­oth­er 4-week Phase III study CF pa­tients with two F508del mu­ta­tions, to the FDA in Q3 and then the EMA in Q4.

Cred­it Su­isse an­a­lysts are ful­ly on board with the plan, say­ing it sig­nals a “clear path for­ward for the triple com­bo, with longer-term da­ta sup­port­ing con­tin­ued ef­fi­ca­cy. We ex­pect ap­proval in 1H20, al­low­ing the co. to treat 90% of CF pts WW. OUS re­im­burse­ment ques­tions re­main.”


Im­age: Ver­tex Phar­ma­ceu­ti­cals

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Pos­i­tive Covid-19 vac­cine da­ta? New mouse study? OWS in­clu­sion? Yep, but some­how, the usu­al tid­bits from In­ovio back­fire

You don’t go more than 40 years in biotech without ever getting a product to market unless you can learn the art of writing a promotional press release. And Inovio captures the prize in baiting the hook.

Tuesday morning Inovio, which has been struggling to get its Covid-19 vaccine lined up for mass manufacturing, put out a release that touched on virtually every hot button in pandemic PR.

There was, first and foremost, an interim snapshot of efficacy from their Phase I program for INO-4800.

Look­ing for 'ex­ter­nal in­no­va­tion,' Boehringer In­gel­heim re­serves $500M+ for new Shang­hai hub

Now that Boehringer Ingelheim’s bet on contract manufacturing in China has paid off, the German drugmaker is anteing up more to get into the research game.

Boehringer has set aside $507.9 million (€451 million) for a new External Innovation Hub to be built in Shanghai over five years. The site will become one of its “strategic pillars” as the team strives to get 71 approvals — either for new products or indications — by 2030, said Felix Gutsche, president and CEO of Boehringer Ingelheim China.

Vas Narasimhan, Novartis CEO (Patrick Straub/​EPA-EFE/​Shutterstock)

No­var­tis pays $678M for kick­back scheme as Vas Narasimhan tries to dis­tance phar­ma gi­ant from shady be­hav­ior

Novartis has reached another large settlement to resolve misconduct allegations, agreeing to pay more than $678 million to settle claims that it had spent hundreds of millions of dollars on lavish dinners, so-called speaking fees and expensive alcohol “that were nothing more than bribes” to get doctors to prescribe Novartis medications.

The top-shelf alcohol and lavish meals included a $3,250 per person night at Nobu in Dallas, a $672-per person dinner at Washington DC’s Smith & Wollensky and a $314 per person meal at Sushi Roku in Pasadena, according to the Justice Department complaint. There were at least 7 trips to Hooters and fishing trips in Alaska and off the Florida coast. Each of these events were supposed to be “speaker programs” where doctors educated other doctors on a drug, but the DOJ alleged many were “bogus” wine-and-dine events where the drug was barely mentioned, if at all.  (“Nobody presented slides on the fishing trips,” the complaint says.)