DB­V's peanut al­ler­gy patch is fi­nal­ly un­der FDA re­view; Rit­ter swal­lows bit­ter pill to ex­plore strate­gic al­ter­na­tives

→ DBV Tech­nolo­gies $DB­VT, which with­drew its ap­pli­ca­tion to mar­ket its peanut al­ler­gy patch late last year, es­sen­tial­ly en­abling ri­val Aim­mune $AIMT to leapfrog it, on Fri­day an­nounced that the FDA has ac­cept­ed its lat­est BLA fil­ing. The agency is ex­pect­ed to make its de­ci­sion by Au­gust 5 — but will host an ad­vi­so­ry pan­el meet­ing to dis­cuss the im­munother­a­py.

Rit­ter Phar­ma­ceu­ti­cals $RT­TR did its best to make lemon­ade with the lemons it was dealt — by vault­ing its lac­tose in­tol­er­ance ther­a­py to­ward a con­fir­ma­to­ry tri­al, de­spite miss­ing the main goal in the pre­ced­ing Phase II/III study. Last month, the com­pa­ny re­port­ed its ther­a­py, RP-G28, failed not on­ly to se­cure a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment ver­sus place­bo in re­duc­ing lac­tose in­tol­er­ance symp­toms, but pa­tients in the place­bo group ac­tu­al­ly did bet­ter than the drug arm. Now, the LA-based com­pa­ny has ap­point­ed a fi­nan­cial ad­vi­sor to ex­plore strate­gic al­ter­na­tives.

As­traZeneca-part­nered French biotech, In­nate Phar­ma, which an­nounced plans for a US IPO worth up to $100 mil­lion a few weeks ago, said to­day that it plans to is­sue and sell about 10.7 mil­lion in an of­fer­ing, to de­vel­op their lead prod­uct, mon­al­izum­ab; fund their mid-stage tri­al of IPH4102 for pa­tients with Sézary syn­drome, MF and PT­CL; and the phase I/II de­vel­op­ment of IPH5401 for sol­id tu­mors, in­clud­ing NSCLC and HCC.

→ The FDA has green­light­ed Pfenex’s ap­pli­ca­tion to mar­ket PF708 to treat os­teo­poro­sis in cer­tain pa­tients at high risk of frac­ture. The ther­a­peu­tic is sim­i­lar to Am­gen’s $AMGN block­buster For­teo, which is made us­ing a type of parathy­roid hor­mone called teri­paratide.

Al­though the FDA plans to des­ig­nate cer­tain prod­ucts like in­sulins and hor­mones from drugs to bi­o­log­ics next year, teri­paratide is not on that list. There­fore, Pfenex sub­mit­ted a 505(b)(2) New Drug Ap­pli­ca­tion for PF708 rather than an ab­bre­vi­at­ed Bi­o­log­ics Li­cense Ap­pli­ca­tion. “We be­lieve PF708 has the po­ten­tial to sig­nif­i­cant­ly en­hance pa­tient ac­cess to an im­por­tant ther­a­py as a cost-ef­fec­tive al­ter­na­tive to For­teo, which had $1.6 bil­lion in glob­al sales in 2018,” said Eef Schim­melpen­nink, CEO of Pfenex. Pfenex is al­so con­duct­ing a sep­a­rate study with the goal of get­ting PF708 des­ig­nat­ed as ther­a­peu­ti­cal­ly equiv­a­lent to For­teo.

Adap­tive Phage Ther­a­peu­tics (APT), a com­pa­ny fo­cused on the de­vel­op­ment of ther­a­pies to com­bat the rise of mul­ti-drug re­sis­tant (MDR) path­o­gen­ic bac­te­ria, has closed its over­sub­scribed, non-bro­kered fi­nanc­ing — rais­ing about $7 mil­lion in pro­ceeds. In­vestors in­clud­ed Alexan­dria Ven­ture In­vest­ments. The com­pa­ny said that pro­ceeds will be used to­wards the sup­port of Phase II clin­i­cal stud­ies for its Phage­Bank ther­a­py for an­tibi­ot­ic-re­sis­tant bac­te­r­i­al in­fec­tions.

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

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.

No­var­tis is ax­ing 150 ear­ly dis­cov­ery jobs as CNI­BR shifts fo­cus to the de­vel­op­ment side of R&D

Novartis is axing some 150 early discover jobs in Shanghai as it swells its staff on the drug development side of the equation in China. And the company is concurrently beefing up its investment in China’s fast-growing biotech sector with a plan to add to its investments in local VCs.

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Democratic presidential candidate, U.S. Sen. Elizabeth Warren (D-MA) speaks during the Nevada Democrats' "First in the West" event at Bellagio Resort & Casino on November 17, 2019 in Las Vegas, Nevada (Getty Images)

Eliz­a­beth War­ren pro­pos­es us­ing com­pul­so­ry li­cens­ing, an­titrust ac­tions to break bio­phar­ma’s con­trol of drug pric­ing — and here are the block­busters she’s tar­get­ing first

Nancy Pelosi’s drug pricing bill may have sparked some industrial strength headaches on the money side of biopharma, but Elizabeth Warren seems determined to become biopharma’s Nightmare on Pennsylvania Avenue.
Warren, one of the top-ranked candidates for the Democratic presidential nomination backing Medicare for all, is circulating a new plan that promises to break the industry’s grip on drug prices — and she has some very specific examples of how she would do it.
The Warren plan would rely on the federal government’s compulsory licensing powers to seize the IP of blockbuster drugs like Truvada and Harvoni to provide them at a fraction of what Gilead sells them for in the US. And she would throw some antitrust actions in as needed to rein in the price of Humira, AbbVie’s cash cow that continues to dominate the list of the most profitable therapeutics on the market.
Notably, she plans to rely on the powers already vested in the federal government, rather than suggest remedies that would require the assent of a deeply divided Congress.
In addition to the blockbusters on the list, Warren sends a clear signal that the same tactics would be used to beef up the supply of cheap antibiotics, as needed. And the same action could befall any other therapy patients can’t afford.

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Mer­ck’s $1B cash gam­ble pays off with a sur­pris­ing PhI­II car­dio suc­cess for Bay­er’s heart drug veri­ciguat

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.

Alk­er­mes forges $950M biotech buy­out deal in a bold bet on an ear­ly-stage CNS drug plat­form

Alkermes $ALKS is investing $100 million cash and committing up to $850 million more in milestones in a big wager on a very early-stage CNS discovery platform. And the biotech is adding $20 million more to fund next year’s new research work on the platform it’s acquiring in today’s buyout with an eye to expanding the research work in oncology.

The biotech, helmed by Richard Pops, is buying Rodin Therapeutics, which had focused early on Alzheimer’s disease. Pops’ buyout, though, isn’t focused solely on the most troublesome sector in pharma R&D.

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(Image: Associated Press)

Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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Left to right: Arthur Pappas, Robert Nelsen, Peter Kolchinsky Doug Cole and David Beier

In rare po­lit­i­cal for­ay, top biotech in­vestors urge Con­gress to re­ject drug pric­ing bill

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. 

Dicer­na scores broad, 'rest of liv­er' deal with No­vo Nordisk, bag­ging $225M in cash to hit some 30 tar­gets with RNAi plat­form

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 wins the in­side track at EMA; PPD files for IPO

→ 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.