Grace Colón, InCarda CEO

With its eyes set on an IPO raise, In­Car­da un­rolls mid-stage da­ta back­ing in­haled ar­rhyth­mia drug

Work­ing to craft an old ar­ry­th­mia drug in­to a more user-friend­ly in­haled for­mu­la­tion, In­Car­da is ex­pect­ing to use new Phase II da­ta re­vealed Wednes­day as the foun­da­tion of a po­ten­tial IPO raise.

The pro­gram, re­pur­pos­ing the oral and IV drug fle­cainide in­to an in­halant, com­plet­ed the lat­est co­hort of its Phase II study with 50% of pa­tients suc­cess­ful­ly con­vert­ing their ir­reg­u­lar heart­beat episodes back to nor­mal rhythm with­in 90 min­utes, the San Fran­cis­co biotech said. That end­point will be the pri­ma­ry goal in a piv­otal Phase III study, ex­pect­ed to be­gin by the end of Sep­tem­ber.

And In­Car­da be­lieves the da­ta are strong enough to get a pos­si­ble IPO ready, as the biotech hired a CFO in con­junc­tion with the news. CEO Grace Colón told End­points News that In­Car­da is in the midst of rais­ing funds for a $70 mil­lion crossover round, and will ex­plore a jump to Nas­daq “should it make sense.”

So far in 2021, 65 biotechs have filed or priced their IPOs, ac­cord­ing to the End­points tal­ly.

Wednes­day’s da­ta come from a study eval­u­at­ing the fle­cainide neb­u­liz­er in a med­ical­ly su­per­vised set­ting, look­ing at in­di­vid­u­als with re­cent-on­set parox­ys­mal atri­al fib­ril­la­tion, or PAF. Pa­tients have been clam­or­ing for new treat­ments, Colón said, be­cause they typ­i­cal­ly re­ceive rate-con­trol­ling treat­ments in emer­gency rooms or oth­er set­tings when they ex­pe­ri­ence PAF episodes, rather than rhythm-con­trol­ling drugs.

“Things with high heart rate like 120 to 160 beats per minute, that’s just fast heart rate pal­pi­ta­tions,” Colón told End­points. “But they al­so ex­pe­ri­ence skipped beats and dizzi­ness, like they can’t breathe. When their heart is not beat­ing nor­mal­ly, they can get se­vere anx­i­ety. It’s a spe­cif­ic type of symp­toms.”

The on­ly cur­rent­ly ap­proved drug to treat these episodes, in­tra­venous ibu­tilide, on­ly has a 90-minute con­ver­sa­tion rate of 38% to 40%, In­Car­da says. Not on­ly did In­Car­da’s pro­gram hit the 50% mark in the Phase II study, but Colón said the mean time of con­ver­sion was just eight min­utes.

Ad­di­tion­al­ly, 72% of those whose heart­beats re­turned to nor­mal rhythm did so with­in the first half hour af­ter treat­ment.

Colón ex­pects much of the new crossover to be di­rect­ed to­ward the Phase III study, which is seek­ing to en­roll 400 pa­tients. Re­searchers are plan­ning to ran­dom­ize pa­tients to the drug arm at a 3-to-1 ra­tio with that 90-minute con­ver­sion rate as the pri­ma­ry, and will al­so check out a host of sec­ondary end­points such as re­lief of symp­toms, hos­pi­tal­iza­tions and time to dis­charge, among oth­ers.

In­Car­da is al­so con­duct­ing stud­ies for the drug in a self-ad­min­is­tered set­ting, with a cur­rent­ly on­go­ing Phase II, and is work­ing on a tri­al for re­peat use. Ini­tial ob­ser­va­tions in­di­cate that pa­tients who re­spond to their first use of the can­di­date be­come “very like­ly” to have a sec­ond re­sponse should an­oth­er episode pop up, Colón said.

There’s been lots of cul­tur­al aware­ness around this is­sue, she added, help­ing dri­ve In­Car­da’s re­search for­ward. Heart rate and rhythm track­ing tech­nol­o­gy has sprout­ed up every­where in wear­able prod­ucts like the Ap­ple Watch, mak­ing it even eas­i­er for pa­tients to keep track of their PAF episodes. In­Car­da has had ear­ly dis­cus­sions with com­pa­nies de­vel­op­ing some of these prod­ucts and will con­tin­ue look­ing for a part­ner should the piv­otal tri­al work out.

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

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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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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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.

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.

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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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.

Alzheimer’s drug bites the dust; Re­struc­ture, re­struc­ture, re­struc­ture; Land­mark di­a­betes OK; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Being in the news business can give one a warped sense of time — it feels like quite a while since we published some of these stories below. But next Saturday’s Endpoints Weekly will definitely be shorter, as we take off Thursday and Friday for Thanksgiving. We will still have the abbreviated edition in your inbox at the usual time.

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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.

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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