Intellia doses first patient with anti-HAE gene therapy; Sobi announces new R&D chief

Gene ther­a­py biotech In­tel­lia Ther­a­peu­tics has dosed its first pa­tient with genome edit­ing can­di­date NT­LA-2002, its drug can­di­date for hered­i­tary an­gioede­ma, a rare ge­net­ic con­di­tion that caus­es swelling un­der the skin. The drug can­di­date has been un­der de­vel­op­ment as a sin­gle-dose gene ther­a­py to pre­vent HAE at­tacks by in­ac­ti­vat­ing the tar­get gene kallikrein B1 (KLKB1). That would, In­tel­lia hopes, re­duce plas­ma kallikrein ac­tiv­i­ty and thus pre­vent HAE at­tacks.

‘With the progress of our first-in-hu­man clin­i­cal study eval­u­at­ing NT­LA-2002 for peo­ple liv­ing with HAE, we look for­ward to be­gin­ning clin­i­cal test­ing as we aim to de­vel­op a sin­gle-dose treat­ment for these pa­tients,’ said In­tel­lia pres­i­dent and CEO John Leonard.

In­tel­lia has been mov­ing full speed ahead over the last six months af­ter bag­ging a stag­ger­ing $600 mil­lion cash raise in June and mak­ing an oc­u­lar CRISPR pact with Spar­ingVi­sion, get­ting a 10% stake with rights to about $200 mil­lion in mile­stones per prod­uct and po­ten­tial roy­al­ties in the process.

So­bi an­nounces new head of R&D and CMO — spoil­ers: It’s one of the di­rec­tors Swedish biotech So­bi is swap­ping out its head of R&D. The biotech an­nounced this morn­ing that one of its mem­bers on their board of di­rec­tors, An­ders Ull­man, will be­come the new R&D head and CMO.

Ull­man will re­place Ravi Rao, a GSK vet­er­an who spent 7 years there be­fore go­ing over to Ae­glea Bio­Ther­a­peu­tics as their CMO in 2019, then join­ing So­bi on­ly last year. Ull­man start­ed out at AZN in the ear­ly 90s be­fore go­ing to Bay­er and Take­da and oth­er places be­fore end­ing up on So­bi’s board just a few months ago.

‘We wel­come An­ders in his new ca­pac­i­ty hav­ing served in 2021 on the So­bi Board of Di­rec­tors,’ said So­bi CEO Gui­do Oelk­ers.

So­bi an­tic­i­pates that the switch will be com­plete some­time ear­ly next year fol­low­ing a han­dover process.

Mer­ck is­sues $1 bil­lion bond as part of $8B pub­lic of­fer­ing, fo­cus­ing on cor­po­rate ESG goals

Mer­ck just is­sued a $1 bil­lion sus­tain­abil­i­ty bond, part of an $8 bil­lion un­der­writ­ten pub­lic of­fer­ing of notes that closed on Fri­day.

This mas­sive bond is in line with Mer­ck’s en­vi­ron­men­tal, so­cial and gov­er­nance (ESG) goals, and ac­cord­ing to Mer­ck CFO Car­o­line Litch­field, the bond is a mas­sive step to­wards in­te­grat­ing ESG in­to the busi­ness.

Mer­ck in­tends to use the net pro­ceeds from this bond of­fer­ing to sup­port projects and part­ner­ships in the com­pa­ny’s pri­or­i­ty ESG ar­eas, and ac­cord­ing to a com­pa­ny state­ment, these in­clude:

Ac­cess to es­sen­tial ser­vices – health care, such as med­i­cines and vac­cines;

In­fec­tious dis­ease re­search and de­vel­op­ment that in­cludes an­timi­cro­bial re­sis­tance, ne­glect­ed and emerg­ing dis­eases;

Re­new­able en­er­gy gen­er­a­tion projects such as new on­site or off­site so­lar or wind gen­er­at­ing ca­pac­i­ty, as well as elec­tric­i­ty gen­er­at­ed from re­new­able sources;

En­er­gy ef­fi­cien­cy ex­pen­di­tures re­lat­ed to the com­pa­ny’s op­er­a­tions, such as en­er­gy-ef­fi­cient heat­ing, ven­ti­la­tion, air con­di­tion­ing, re­frig­er­a­tion, light­ing, roof­ing or elec­tri­cal equip­ment, en­er­gy mon­i­tor­ing, con­trol so­lu­tions and en­er­gy as­sess­ments, in­clud­ing smart me­ters and con­trol au­toma­tion de­vices; and

Pol­lu­tion pre­ven­tion and con­trol projects to re­duce and man­age emis­sions to air or wa­ter, as well as re­cy­cling projects and ef­forts to di­vert non-haz­ardous and/or haz­ardous waste away from land­fills.

Mer­ck said in a state­ment that they have com­mit­ted to an­nu­al re­port­ing on the al­lo­ca­tion of the pro­ceeds of the bond, along with the re­main­ing bal­ance of un­al­lo­cat­ed pro­ceeds.

Sensor-based technology for clinical trial data collection represents the latest medical paradigm shift. There are more than 700 clinical studies involving wearable devices currently underway in the United States. A study from Intel IT projects their inclusion in clinical trials will surge to 70% by 2025.

Apps, biosensors and patient-centered technologies increase visibility of comprehensive patient data. Pharma leaders anticipate the benefits of wearables to include better data (58%), faster results (33%) and lower trial costs (10%).

When Bristol Myers Squibb celebrated the approval of ozanimod — branded Zeposia — in ulcerative colitis earlier this year, the company touted the first gastrointestinal indication for an S1P receptor modulator.

Now Pfizer wants to give the pharma rival a run for its money.

Pfizer is dropping $6.7 billion to acquire Arena Pharmaceuticals, whose lead drug, etrasimod, targets the sphingosine 1-phosphate receptor.

Unlock this story instantly and join 125,400+ biopharma pros reading Endpoints daily — and it’s free.

Rumors have swirled around a potential buyout of Switzerland’s Vifor Pharma by Australia’s CSL since the start of December, and now the gossip reportedly has some truth to it.

Vifor confirmed to Reuters early Monday that it is in discussions to be acquired by CSL for more than $8.5 billion, sending its shares up more than 15% in overseas trading. The deal is expected to be finalized Tuesday, according to Australian media, with one large investor reportedly willing to pay more than $173 per share — about $60 more than Vifor’s price before the rumors began circulating on Dec. 2.

Unlock this story instantly and join 125,400+ biopharma pros reading Endpoints daily — and it’s free.

AbbVie’s Migraine Relief-Tok on TikTok

Relaxation apps have nothing on AbbVie migraine med Ubrelvy’s TikTok. Ocean waves and birds chirping offer respite and a quiet scrolling break on the more typically frenetic social media app.

AbbVie calls it Migraine Relief-Tok and in a series of ads feature different calming sounds of nature with the advice: ‘Take a Break. Breathe in, Breathe out.’

The unexpected quiet in what can be an overstimulating experience of music, dancing, pop culture and politics is intentional. AbbVie knows that like many people, the 40 million people who suffer from migraine, are also on social media and TikTok.

Unlock this story instantly and join 125,400+ biopharma pros reading Endpoints daily — and it’s free.

In the face of short attacks, it is not uncommon these days for companies to cry foul and defend themselves against what they call false and misleading statements. But are these short sellers involved in something more nefarious? The Department of Justice is reportedly trying to find out through a sweeping criminal investigation.

Federal investigators are scrutinizing an unknown number of hedge funds and research firms — alongside their trading of at least several dozen shorts — to see if any kind of deception or manipulation was involved, Reuters and Bloomberg reported.

Roberto Bellini, Bellus Health CEO

Do investors want the good news, or the bad news first? Bellus went with the good news, sharing that its chronic cough contender hit the primary endpoint in a Phase IIb trial, lining it up for a Phase III study in the second half of next year where it could pose stiff competition for Merck.

Amidst all the commotion, the Canadian biotech also revealed that the same candidate flunked a proof-of-concept trial in atopic dermatitis, and the company will now steer the program away from pruritic conditions.

Unlock this story instantly and join 125,400+ biopharma pros reading Endpoints daily — and it’s free.

Soon after San Francisco-based Genentech won an EUA for tocilizumab as a treatment for hospitalized Covid patients last summer, the company announced a shortage of the drug while pointing to the emergence of the Delta variant and the slowing of vaccination rates across the US.

‘This new wave of the pandemic has led to Genentech experiencing an unprecedented demand for Actemra IV– well-over 400% of pre-COVID levels over the last two weeks alone and it continues to increase,’ the company said in August.

Merck’s potential Covid-19 treatment molnupiravir will not be used in France, French regulators said Friday.

The French National Authority of Health cited the potential impact of the Omicron variant, the fact that Regeneron’s mAb cocktail is more effective, and the pill’s own lack of efficacy as reasons for denying early access of the drug to patients experiencing mild to moderate cases of Covid-19. France has already pre-ordered hundreds of thousands of the pills, with the goal of treating 50,000 patients.

The Senate Finance Committee on Saturday released the latest text of President Joe Biden’s $2 trillion spending package, paid for at least in part with new negotiating power for Medicare and inflation rebates drugmakers will have to pay if their drug prices rise too quickly each year.

But now, generic drugs at risk of shortage and biosimilars have been cut out of the rebates, as their industry lobbying groups had sought. They’d said the inclusion of such rebates and negotiations could increase the likelihood of drug shortages and create barriers to competition.
https://endpts.com/intellia-doses-first-patient-with-anti-hae-gene-therapy-sobi-announces-new-rd-chief/