→ The EMA of­fi­cial­ly said good­bye to its Ca­nary Wharf head­quar­ters in Lon­don on Fri­day, to set up shop in Am­s­ter­dam — a de­ci­sion trig­gered by Britain’s 2016 de­ci­sion to leave the EU.

To­day we are clos­ing our Lon­don of­fices af­ter al­most a quar­ter of a cen­tu­ry. Thank you, Lon­don for be­ing such a gra­cious host! https://t.co/R8LXjfzvp3 pic.twit­ter.com/8abqM­cbROJ

— EU Med­i­cines Agency (@EMA_News) March 1, 2019

→ The EU reg­u­la­tor al­so is­sued pos­i­tive rec­om­men­da­tions for a slate of treat­ments, list­ed be­low:

EMA’s hu­man med­i­cines com­mit­tee rec­om­mends 8 #med­i­cines for ap­proval at its Feb­ru­ary 2019 meet­ing https://t.co/VCEd­e­qi­Aar #CHMP pic.twit­ter.com/0Z1cC­N­fkng

— EU Med­i­cines Agency (@EMA_News) March 1, 2019

• Sanofi/Re­gen­eron‘s Dupix­ent for se­vere asth­ma, set­ting up the ex­pan­sion of the med­i­cine’s la­bel, which cur­rent­ly cov­ers atopic der­mati­tis
• Sanofi $SNY had an­oth­er en­dorse­ment, for its di­a­betes drug so­tagliflozin, to be sold un­der the brand name Zyn­quista
• Bio­Marin Phar­ma­ceu­ti­cals $BM­RN has won back­ing for peg­valiase, which has been dubbed Pa­lynz­iq, for phenylke­tonuria, a rare but po­ten­tial­ly se­ri­ous in­her­it­ed meta­bol­ic dis­ease
• The agency en­dorsed grant­i­ng a con­di­tion­al mar­ket­ing au­tho­riza­tion for Akcea’s volane­sors­en — to be sold as Waylivra — as the first med­i­cine to treat fa­mil­ial chy­lomi­cron­aemia syn­drome, a rare ge­net­ic dis­ease that thwarts the body from break­ing down fats
• An­oth­er con­di­tion ap­proval was rec­om­mend­ed for Por­to­la Phar­ma­ceu­ti­cals’ $PT­LA an­dex­anet al­fa, brand­ed as On­dexxya — an an­ti­dote for pa­tients on cer­tain clot-fight­ers, when the use of the an­ti­co­ag­u­lants leads to life-threat­en­ing or un­con­trolled bleed­ing
• Ab­b­Vie $AB­BV se­cured rec­om­men­da­tion for risankizum­ab — brand­ed as Skyrizi — for plaque pso­ri­a­sis

These rec­om­men­da­tions fol­lowed two oth­ers from Thurs­day:

• GSK’s $GSK zanamivir — to be sold as Dec­to­va— for com­pli­cat­ed and po­ten­tial­ly life-threat­en­ing in­fluen­za
• Pfiz­er $PFE se­cured back­ing for con­di­tion­al ap­proval of lor­la­tinib — brand­ed as Lorvi­qua — to treat a form of non-small cell lung can­cer

→ Un­der fire from Star­board and Welling­ton — which have made their op­po­si­tion against the $74 bil­lion buy­out of Cel­gene $CELG abun­dant­ly clear — Bris­tol-My­ers $BMY has been try­ing to shore up in­vestor sup­port be­hind-the-scenes, CN­BC re­port­ed on Fri­day. The re­port sug­gest­ed Bris­tol had sent ex­ec­u­tives to New York to meet with in­sti­tu­tion­al in­vestors sev­er­al times over the last two weeks and met with in­vestors in Boston on Wednes­day and Thurs­day, cit­ing a per­son briefed on the meet­ings.

→  UK-based AdoRx Ther­a­peu­tics has tied up with J&J $JNJ to de­vel­op lung can­cer drugs in a deal with undis­closed fi­nan­cial terms.

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

More than 3 years after Merck stepped up and paid $1 billion in cold, hard cash to gain the US commercial rights to Bayer’s high-risk heart drug vericiguat in a broad-ranging cardio alliance, the partners say their Phase III study has come through with promising data and a date with regulators.
We don’t have the data, and won’t until they put it out at an upcoming scientific session, but Merck touted the results, saying that their big Phase III VICTORIA study hit the primary endpoint  — with vericiguat combined with available therapies reducing “the risk of the composite endpoint of heart failure hospitalization or cardiovascular death in patients with worsening chronic heart failure with reduced ejection fraction (HFrEF) compared to placebo when given in combination with available heart failure therapies.”
Depending on the hard data, and how it breaks out with the combinations used, this drug could pose a threat to Novartis’ blockbuster drug Entresto, currently at $1.6 billion while analysts expect peak sales to hit $4 billion.
The drug is a soluble guanylate cyclase (sGC) stimulator, which Bayer and Merck have had high hopes for. Evidently, so did cardiologists. Cowen’s last analysis set potential sales at $400 million in 2024, but that number could go up significantly now.
Cowen’s Steve Scala noted this morning:
Vericiguat could be a lucrative product for Merck, and one with potentially under-appreciated value. At Cowen’s Therapeutics Conference in September 2019, 80% of specialists anticipated a positive result from VICTORIA whereas only 51% of investors shared this optimism.
Investigators recruited more than 5,000 patients at more than 600 centers in 42 countries for this study — one of the most expensive propositions in R&D. Millions of people in the US suffer from heart failure with reduced ejection fraction when the failing heart fails to contract properly to eject blood into the system. Bayer holds ex-US rights to the drug and also stands to earn cash from the $1.1 billion in milestones Merck agreed on for their collaboration.
Remarkably, the drug was pushed into Phase III despite failing the mid-stage trial — though investigators flagged a success at the high dose of 10 mg. In VICTORIA, researchers started patients at 2.5 mg and then titrated up to 5 and then 10 mg.

Thirteen of the top biotech venture capitalists in the country wrote a letter last week warning lawmakers that if Congress passes a drug pricing bill House Speaker Nancy Pelosi has put before lawmakers, they won’t be able to invest in biomedical research at their current rate, and patients will suffer.

“If policies such as those included within H.R. 3, the Lower Drug Costs Now Act, are passed, our ability to continue to invest in future biomedical innovation will be severely constrained, thus crushing the hopes of millions of patient waiting for the next breakthroughs to treat or cure their cancers, rare genetic diseases, Alzheimer’s, or other serious and life-threatening conditions,” they wrote in a letter addressed to the highest-ranking Democrats and Republicans in the House and Senate and acquired by Endpoints News. 

Turns out Dicerna wasn’t done with deals yet after locking in $200 million upfront from Roche for a hepatitis B cocktail two weeks ago.

Novo Nordisk has signed on as the latest partner to its GalXC RNAi platform, handing over $175 million in cash to claim any and all targets of interest in liver-related cardio-metabolic diseases that are not already reserved in previous pacts. The Danish drugmaker — which has signaled its interest to expand considerably beyond its core diabetes franchise into areas like NASH — is also purchasing $50 million worth of Dicerna’s equity at a 25% premium of $21.93 per share. More research payments and milestones extending to the billions are on the line.

→ Gene therapy maker Orchard Therapeutics has been granted an accelerated assessment for OTL-200 by the EMA’s Committee for Medicinal Products for Human Use (CHMP). The gene therapy — in development in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy — being used towards the treatment of metachromatic leukodystrophy.

→ Pharmaceutical Product Development has announced that its parent company, PPD, Inc has submitted a draft to the SEC relating to the proposal of an IPO of the parent company’s common stock. Number of shares and price range have not yet been determined.

In the story lawmakers and drug pricing reform advocates have told about the drug industry, there are perhaps few greater villains than Humira and its maker AbbVie.

Between 2012 and 2018, AbbVie upped the drug’s annual after-rebates cost from $19,000 to $38,000 in the US, with sticker prices now over $60,000 per year — increases that led to accusations of price gouging, most recently from Democratic presidential frontrunner Elizabeth Warren.