→ Pen­ny stock North­west Bio­ther­a­peu­tics is still hang­ing on. The com­pa­ny, which is de­vel­op­ing DC­Vax for sol­id tu­mor can­cers, has an­nounced that they’ve reached a set­tle­ment with the SEC. The com­pa­ny has agreed to pay a fine of $250,000 “in con­nec­tion with past weak­ness­es in its in­ter­nal con­trols” and the com­pa­ny will nei­ther ad­mit nor de­ny any vi­o­la­tions un­der the set­tle­ment.

→ Long-slid­ing biotech Five Prime Ther­a­peu­tics will lay­off 70 em­ploy­ees as part of a cor­po­rate re­struc­tur­ing as it strug­gles to find its foot­ing. The on­col­o­gy-fo­cused com­pa­ny has been in free-fall since late 2017, when its Bris­tol-My­ers Squibb-part­nered drug cabi­ral­izum­ab showed weak ef­fi­ca­cy and a trou­bling safe­ty pro­file in ear­ly-stage pan­cre­at­ic can­cer. In Jan­u­ary, they cut 41 jobs, about a fifth of its work­force at the time, and in Sep­tem­ber they tapped William Ringo as in­ter­im CEO af­ter Aron Knicker­bock­er re­signed. On Thurs­day, Ringo an­nounced the lay­offs. “The com­pa­ny will re­tain a small re­search group fo­cused on ad­vanc­ing three whol­ly-owned, late-stage re­search as­sets and will in­creas­ing­ly re­ly on out­sourc­ing and con­tract­ed ca­pa­bil­i­ties,” the com­pa­ny wrote.

→ Is­raeli biotech Ther­a­pix Bio­sciences — a com­pa­ny that is in­ves­ti­gat­ing the use of cannabi­noid-based drugs in the re­duc­tion of symp­toms in adults suf­fer­ing from Tourette’s syn­drome — has re­vealed up­dates on its planned merg­er with Des­tiny Bio­sciences. Ac­cord­ing to the let­ter of in­tent, “the par­ties agreed to al­low un­til Oc­to­ber 31, 2019, to com­plete the de­fin­i­tive agree­ment be­fore aban­don­ing the trans­ac­tion.” Ther­a­pix’s board of di­rec­tors said that it is will­ing to give Des­tiny ad­di­tion­al time to reach a de­ci­sion. Des­tiny is cur­rent­ly car­ry­ing out a val­u­a­tion analy­sis of the merg­er.

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.