Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

Nick Leschly Linkedin

Er­rant Gene Ther­a­peu­tics was for­mal­ly es­tab­lished in Oc­to­ber 2003 by Patrick Giron­di, whose son Roc­co was two years old when he was di­ag­nosed with a se­vere form of the rare, in­her­it­ed blood dis­or­der. In 2000, Er­rant con­tact­ed an MSK sci­en­tist, Michel Sade­lain, who had pub­lished a pa­per ex­hibit­ing the po­ten­tial of his gene ther­a­py tech­nol­o­gy in mice — and be­gan to fund Sade­lain’s re­search. Five years lat­er, Er­rant pur­chased from MSK the ex­clu­sive, glob­al rights to har­ness Sade­lain’s tech­nol­o­gy to work on gene ther­a­pies for blood dis­or­ders: be­ta-tha­lassemia and sick­le cell dis­ease. In the years that fol­lowed, Er­rant worked with MSK to cre­ate a vec­tor — which was then patent­ed by the com­pa­ny in the Unit­ed States in 2009,  the law­suit said.

Michel Sade­lain Can­cer Re­search In­sti­tute

In late 2016, Er­rant al­leged it dis­cov­ered that blue­bird and Third Rock — a firm which Leschly was a found­ing mem­ber and a for­mer part­ner of — were in ca­hoots to ob­struct its gene ther­a­py from reach­ing the mar­ket by en­gag­ing in a de­lib­er­ate pat­tern of fraud and de­cep­tion, and in­dica­tive of “a trou­bling pat­tern of tor­tious busi­ness prac­tices un­der­tak­en by phar­ma­ceu­ti­cal com­pa­nies to se­cret­ly and sys­tem­at­i­cal­ly ‘kill’ com­pet­ing med­ical treat­ments.”

Back in 2010, Er­rant was ready to ini­ti­ate clin­i­cal tri­als with MSK and the NIH. At the time, the oth­er drug de­vel­op­er with an ex­per­i­men­tal tha­lassemia gene ther­a­py was Genetix Phar­ma­ceu­ti­cals. That year, Third Rock in­vest­ed in Genetix, in­stalled Leschly as chief, and switched the com­pa­ny’s name to blue­bird bio.

Ac­cord­ing to the law­suit, Third Rock then met with MSK and “ad­mit­ted” that the Er­rant vec­tor out­shined blue­bird’s vec­tor — and was ad­vised to ap­proach Er­rant about buy­ing its tech­nol­o­gy. When Third Rock did not agree to aban­don the blue­bird vec­tor in fa­vor of Er­rant’s, the plain­tiff as­sert­ed that blue­bird and Third Rock moved to snuff the de­vel­op­ment of the Er­rant vec­tor by ac­cess­ing the tech­nol­o­gy via a “se­cret agree­ment” with MSK. In ad­di­tion, blue­bird and Third Rock re­lied up­on their re­la­tion­ships with new­ly crowned MSK chief Craig Thomp­son to fur­ther stall the de­vel­op­ment of Er­rant’s gene ther­a­py, the law­suit added.

Craig Thomp­son MSK

Lat­er in 2010, MSK de­mand­ed that Er­rant find a com­mer­cial part­ner as a con­di­tion be­fore start­ing clin­i­cal tri­als — but de­spite Er­rant ful­fill­ing that con­di­tion, MSK re­ject­ed the con­tract and in­stead ini­ti­at­ed ar­bi­tra­tion against Er­rant, the drug de­vel­op­er claimed in the law­suit. Even­tu­al­ly by 2011, Third Rock and MSK “con­vinced” Er­rant to give MSK its vec­tor in ex­change for a 50% roy­al­ty for any prod­uct de­vel­oped us­ing it, Er­rant said, sug­gest­ing it is owed di­rect dam­ages to the tune of “hun­dreds of mil­lions of dol­lars.”

A Third Rock spokesper­son said it had no com­ment, while a blue­bird spokesper­son said the com­pa­ny does not com­ment on lit­i­ga­tion.

This is not the first time Er­rant’s Giron­di has lashed out at MSK. A re­port by the New York Times in 2015 chron­i­cles Giron­di’s pre­vi­ous at­tempt. MSK was forced to take back con­trol of the project be­cause Giron­di — a high school dropout who was ar­rest­ed var­i­ous times in his youth, but dodged prison by join­ing the Air Force — lacked the”nec­es­sary mon­ey and the ex­per­tise,” the re­port said, cit­ing An­drew Maslow, the di­rec­tor of in­dus­tri­al af­fairs at MSK un­til ear­ly 2012.

End­points News has con­tact­ed MSK for com­ment.

Ear­li­er this month, blue­bird’s Zyn­te­glo — a one-shot gene ther­a­py en­gi­neered to re­place a life­time of trans­fu­sions and med­ica­tion for pa­tients aged 12 years and old­er — was ap­proved in Eu­rope. While the Cam­bridge, Mass­a­chu­setts-based com­pa­ny has pro­duced per­sua­sive da­ta — dra­mat­i­cal­ly re­duc­ing the need for blood trans­fu­sions in a 22-pa­tient study — the gene ther­a­py has fall­en short of a cure in se­vere pa­tients. Phase III tri­als de­signed to con­firm Zyn­te­glo’s ben­e­fit are on­go­ing, and US ap­proval is an­tic­i­pat­ed next year.

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.