On a roll, Ver­tex racks up im­pres­sive PhI­II da­ta for VX-659 com­bos in cys­tic fi­bro­sis

Ver­tex $VRTX has come up with a stack of pos­i­tive Phase III da­ta points for their cys­tic fi­bro­sis triple in­clud­ing the ex­per­i­men­tal VX-659. And its R&D group says they can ride these re­sults all the way to an FDA ap­proval.

Right now we’re just get­ting the pri­ma­ry end­point read­out, which is im­pres­sive. VX-659 is be­ing cred­it­ed with a 14% place­bo-ad­just­ed im­prove­ment from base­line in FEV1 for pa­tients with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion, with a clear score on the p val­ue (p<0.0001). 

In a sep­a­rate Phase III pa­tients ho­mozy­gous for the F508del mu­ta­tion al­ready tak­ing Symdecko saw an av­er­age 10% im­prove­ment with VX-659 added on.

Resh­ma Ke­wal­ra­mani

Now Ver­tex CMO Resh­ma Ke­wal­ra­mani says they will wait for the Phase III num­bers on their oth­er big add-on, VX-445, in Q1 be­fore de­cid­ing on the reg­u­la­to­ry strat­e­gy as they look to ex­pand their hold on CF.

Of par­tic­u­lar note: These new da­ta points are right in line with Phase II re­sults, of­fer­ing some clear par­al­lel sup­port for their com­bi­na­tion to take to reg­u­la­tors. 

The da­ta “live up to the hype,” not­ed Stifel’s Paul Mat­teis, high­light­ing the close com­par­i­son be­tween the last two tri­als.

While de­tails (as ex­pect­ed) are lim­it­ed be­yond the pri­ma­ry end­point, from press re­lease there’s noth­ing to nit­pick (in our view) for what has been reaf­firmed as a trans­for­ma­tion­al drug reg­i­men in “het-min” CF pa­tients, a pop­u­la­tion that rep­re­sents a mul­ti-bil­lion dol­lar op­por­tu­ni­ty with no tar­get­ed ther­a­pies ap­proved to­day.

Michael Yee at Jef­feries al­so joined the cheer­ing sec­tion.

In ho­mozy­gous which is a mar­ket al­ready well pen­e­trat­ed by Orkam­bi and Symdeko, this triple of ‘659 is meant to sig­nif­i­cant­ly im­prove FEV ef­fi­ca­cy from a cur­rent +4% to +14%, which is a huge im­prove­ment and ben­e­fit for pa­tients and will even­tu­al­ly re­place Orkam­bi/Symdeko over time and cre­ates a huge bar­ri­er to en­try giv­en the over­all ef­fi­ca­cy/safe­ty pro­file of ‘659 in this pop­u­la­tion.

Those views helped Ver­tex shares gain about 4% in ear­ly trad­ing Tues­day.

Ver­tex has been sur­round­ed by ri­vals look­ing to carve off a piece of the block­buster mar­ket that the Boston biotech has been carv­ing out for it­self. In sev­er­al cas­es, we’ve seen com­peti­tors stum­ble with mixed or poor da­ta. Pro­teosta­sis has man­aged to es­cape that fate, with some pos­i­tive ear­ly-stage num­bers of its own. But Ver­tex has been dom­i­nant in CF for years now, and ap­pears de­ter­mined to stay well in the lead.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Isao Teshirogi, Shionogi president and CEO (Kyodo via AP Images)

Sh­ionogi's Covid an­tivi­ral lands first ap­proval in Japan's new emer­gency ap­proval path­way

Japanese regulators on Tuesday signed off on Shionogi’s homegrown antiviral for Covid-19, known as Xocova (ensitrelvir), making it the first approval under Japan’s emergency regulatory approval system.

The emergency approval, following a back-and-forth with regulators since last February, is based on a safety profile with more than 2,000 patients who have accessed the pill, and clinical symptomatic efficacy for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in patients with mild to moderate SARS-CoV-2 infection, regardless of risk factors or vaccination status, and during the Omicron-dominant phase of the pandemic.

Karen Aiach, Lysogene CEO (RE(ACT) Discovery Institute)

Gene ther­a­py flunks PhII/III study, but for­mer Sarep­ta part­ner sees a path for­ward — if it can find the cash

The development path for Lysogene’s gene therapy for MPS IIIA has been a rocky one. After the FDA slapped a partial clinical hold on a Phase II/III study, a patient already dosed in the trial died, although it was deemed unrelated to treatment. Then earlier this year, Sarepta pulled out of their three-year partnership due to disagreements on who will handle commercial supply.

And now, Lysogene reported the trial has failed its primary endpoint.

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Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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