Months ago, Foamix leaned on its biggest share­hold­ers — Per­cep­tive Ad­vi­sors and Or­biMed — to fi­nan­cial­ly grease its wheels, ahead of the FDA de­ci­sion date for its ac­ne ther­a­py. On Fri­day, that ap­proval came in — and the top­i­cal for­mu­la­tion of the an­tibi­ot­ic minocy­cline is set for a Jan­u­ary launch.

The ther­a­py, Amzeeq (for­mer­ly known as FMX101), was ap­proved to treat in­flam­ma­to­ry le­sions of non-nodu­lar mod­er­ate-to-se­vere ac­ne vul­garis in pa­tients aged 9 and old­er.

Minocy­cline, a sec­ond-gen­er­a­tion tetra­cy­cline, was first syn­the­sized in 1967 and made its maid­en en­try in­to the US mar­ket in 1972. It is a broad-spec­trum an­tibi­ot­ic — em­ployed as a weapon across a myr­i­ad of con­di­tions in­clud­ing ac­ne. But its use as an oral for­mu­la­tion has been lim­it­ed in ac­ne due to sys­temic side-ef­fects. Sci­en­tists have strug­gled with en­gi­neer­ing a top­i­cal ver­sion be­cause the com­pound is typ­i­cal­ly un­sta­ble in such tra­di­tion­al for­mu­la­tions.

Foamix’s tech­nol­o­gy plat­form de­liv­ers minocy­cline in a foam for­mu­la­tion. It was test­ed in three piv­otal stud­ies that in­volved 2,418 pa­tients al­to­geth­er.

In the first two stud­ies: tri­al 04 and tri­al 05, a to­tal of 961 sub­jects with mod­er­ate-to-se­vere ac­ne were ran­dom­ized to ei­ther re­ceive the Foamix foam or ve­hi­cle foam once dai­ly for 12 weeks. The twin main goals of each study were: the ab­solute change in the num­ber of in­flam­ma­to­ry le­sions (papules and pus­tules); and the pro­por­tion of pa­tients se­cur­ing an in­ves­ti­ga­tor’s glob­al as­sess­ment (IGA) score of “clear” or “al­most clear” and at least a two-grade im­prove­ment from base­line at week 12.

While the Foamix foam hit sta­tis­ti­cal sig­nif­i­cance on both end­points in tri­al 05, the ther­a­py did not make the cut in the IGA score in tri­al 04 — forc­ing a third late-stage tri­al, which suc­ceed­ed on both counts.

The Is­raeli drug de­vel­op­er has a dif­fer­ent minocy­cline foam for­mu­la­tion, called FMX103, for rosacea un­der FDA re­view — a de­ci­sion is ex­pect­ed by ear­ly June. Mean­while, the com­pa­ny is al­so test­ing a minocy­cline + ada­pa­lene for­mu­la­tion for ac­ne — that com­bi­na­tion is in mid-stage de­vel­op­ment.

Both Amzeeq and the rosacea ther­a­py should see sig­nif­i­cant up­take and gen­er­ate $250 mil­lion-plus in peak sales each, Cowen’s Ken Cac­cia­tore wrote in a note, cit­ing ex­perts in the field.

“As for Amzeeq’s pric­ing, al­though the ex­act fig­ure has not yet been dis­closed, we ex­pect that the po­ten­tial for $200-400 net is rea­son­able and achiev­able, giv­en Foamix’s ini­tial pos­i­tive in­ter­ac­tion with pay­ors and brand­ed pric­ing of oth­er dif­fer­en­ti­at­ed ac­ne op­tions.”

End­points News has con­tact­ed Foamix $FOMX re­gard­ing its Amzeeq pric­ing plans.

The com­pa­ny has been build­ing out its com­mer­cial in­fra­struc­ture ahead of ap­proval, with rough­ly 50+ sales reps fo­cus­ing on 6,000 pre­scribers that cov­er rough­ly 75% of the ac­ne pre­scrip­tion mar­ket, Cac­cia­tore not­ed.

“This spe­cif­ic tar­get­ing will al­low man­age­ment to avoid a very cost­ly launch ap­proach that oth­er com­pa­nies have un­der­tak­en, which has re­sult­ed in over­ly broad, un­fo­cused, all-ter­ri­to­ries mar­ket­ing ef­fort that has of­ten burned sig­nif­i­cant cap­i­tal with poor re­sults. In­stead, Foamix is ex­pect­ed to move re­gion­al­ly – and more thought­ful­ly – ac­cord­ing to the man­aged care cov­er­age tim­ing,” he said.

“Clear­ly small com­pa­ny launch­es have had strug­gles of late, but we be­lieve Foamix is go­ing to thought­ful­ly in­cor­po­rate the learn­ings (and mis­takes) of oth­er prod­uct in­tro­duc­tions, es­pe­cial­ly in terms of the nec­es­sary co-pay sup­port pro­grams. We would al­so note that ‘103 — once ap­proved for rosacea — will tar­get near­ly the ex­act same clin­i­cian base to lever­age the Amzeeq re­la­tion­ship build­ing and com­mer­cial plat­form.”

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.