FDA hands Mor­phoSys and In­cyte a quick OK on their po­ten­tial block­buster CAR-T al­ter­na­tive

Near­ly three years af­ter okay­ing the CAR-Ts Yescar­ta and Kym­ri­ah, the FDA has ap­proved a new CD19 ther­a­py.

Mor­phoSys’ Mon­ju­vi, or taf­a­sita­m­ab-cx­ix, was cleared Fri­day for use in re­frac­to­ry dif­fuse large B-cell lym­phoma (DBLCL). The ap­proval sets up both Mor­phoSys and their com­mer­cial part­ner In­cyte to com­pete with Gilead and No­var­tis in the ul­tra-com­pet­i­tive in­di­ca­tion, where sim­i­lar tri­al re­sults and far eas­i­er de­liv­ery could al­low them to cut a fair share of the mar­ket.

The first ap­proved in­di­ca­tion is in sec­ond-line DBLCL, for pa­tients in­el­i­gi­ble for au­tol­o­gous stem cell trans­plant. Mizuho’s Mara Gold­stein es­ti­mat­ed that mar­ket at around 6,000 US pa­tients and said the drug had “block­buster po­ten­tial,” but she not­ed those pa­tients have sev­er­al oth­er op­tions avail­able to them.

“The ini­tial mar­ket op­por­tu­ni­ty as a sec­ond-line treat­ment … is mean­ing­ful,” she wrote in a note to in­vestors, “though we are some­what cau­tious on the ramp of the drug giv­en the com­pet­i­tive land­scape for this in­di­ca­tion that in­cludes sev­en drug reg­i­mens as well as treat­ment with CAR-T ther­a­py.”

SVB Leerink’s Ge­of­frey Porges called the ap­proval a “best case sce­nario,” not­ing it had been un­clear whether the drug would be ap­proved for sec­ond-line or third-line ther­a­py. He es­ti­mat­ed a po­ten­tial pa­tient pop­u­la­tion around 12,000.

Both an­a­lysts sin­gled out Mon­ju­vi’s du­ra­tion in par­tic­u­lar. The 21.7 months it showed at the top line read­out is more than twice the 6 months to a year Roche’s an­ti­body-drug Po­livy, ap­proved last year, has shown.

“We be­lieve the CAR-T like ef­fi­ca­cy demon­strat­ed by tafa, as well as its fa­vor­able safe­ty pro­file should en­able broad adop­tion in the 2L set­ting,” Porges wrote in a note to in­vestors,  “and ex­pect the prod­uct to be pre­ferred over Roche’s CD79 ADC Po­livy.”

Si­mon Mo­roney

For­mer Mor­phoSys CEO Si­mon Mo­roney po­si­tioned Mon­ju­vi as an op­tion for pa­tients who can’t han­dle the in­tense bod­i­ly stress of high-dose chemother­a­py or stem cell trans­plant, or of CAR-T. In ad­di­tion to the cost­ly and lengthy process of ex­tract­ing, treat­ing and re-im­plant­i­ng T cells, CAR-T ther­a­pies al­so re­quire pa­tients to un­der­go “con­di­tion­ing” reg­i­mens of in­ten­sive chemother­a­py to clear out the bone mar­row and es­sen­tial­ly give a pock­et for the new cells to take hold.

By con­trast, Mon­ju­vi is an an­ti-CD19 an­ti­body that is giv­en in­tra­venous­ly and re­quires no con­di­tion­ing.

“Our pa­tients tend to be old­er — me­di­an age in the tri­al is 72,” Mo­roney told Med­C­i­ty News last June, re­fer­ring to par­tic­i­pants in their piv­otal Phase II study. “They have co­mor­bidi­ties and are not el­i­gi­ble for CAR-T.”

The FDA de­ci­sion is an ac­cel­er­at­ed ap­proval based on re­sults from that Phase II study. Topline re­sults re­leased last year showed a 55% over­all re­sponse rate and a 37% com­plete re­sponse rate. The lat­est da­ta, re­leased in May, showed a 59% re­sponse rate, a 39% com­plete re­sponse rate, and a me­di­an du­ra­tion of re­sponse that had risen to 34.6 months — near­ly 3 years. The com­plete re­sponse rates were in line with the ear­ly CAR-T tri­als, and the FDA grant­ed the drug pri­or­i­ty re­view.

Jean-Paul Kress

Both Mor­phoSys and In­cyte have staked a lot on Mon­ju­vi. Mor­phoSys raised $208 mil­lion in an IPO in 2018, fun­nel­ing much of that cash in­to build­ing out a US com­mer­cial op­er­a­tion in an­tic­i­pa­tion of their lead drug’s ap­proval. They tapped the US com­mer­cial lead for Mer­ck KGaA’s Baven­cio, David Trexler, to lead that ef­fort. And af­ter Mo­roney’s re­tire­ment, Jean-Paul Kress stepped in to lead a new stage for the com­pa­ny.

In­cyte, mean­while, spent $900 mil­lion ear­li­er this year to join with In­cyte on the com­mer­cial launch, hop­ing the CAR-T al­ter­na­tive could pour some oil in­to a sput­ter­ing R&D en­gine. Mizuho’s Gold­stein said the drug would help di­ver­si­fy In­cyte’s rev­enue base, but its over­all im­pact may be mut­ed.

“Mon­ju­vi may not have enough im­pact in the near term to al­ter the fun­da­men­tal pic­ture for the com­pa­ny,” she said of In­cyte.

Gold­stein al­so called the drug “val­i­da­tion” for Xen­cor’s plat­form. The Cal­i­for­nia biotech orig­i­nal­ly de­vel­oped the an­ti­body. The ap­proval trig­gered a $25 mil­lion mile­stone for them.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists

President Donald Trump, who seems intent on announcing a COVID-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

Clay Siegall (Life Science Washington via YouTube)

#ES­MO20: Seat­tle Ge­net­ics eyes 4th ap­proval with new da­ta in a crowd­ed field

Does Seattle Genetics have another approval on its hands?

The last 12 months, not so great for the world, has been great for Seattle Genetics. The company landed two separate FDA approvals, signed a $4.5 billion deal with Merck and watched antibody-drug conjugates — the technology they spent years developing to broad industry skepticism — emerge suddenly as one of the most popular approaches in oncology. And on Monday at ESMO, the company and their partners at Genmab unveiled the data behind the ADC it hopes will provide its next major FDA approval.

Jonathan Rigby, Immune Regulation group CEO

Im­mune Reg­u­la­tion, tak­ing two clin­i­cal pro­grams to 're­set' the im­mune sys­tem, nets $53M+ Se­ries B

A little under two years after a company rebranding, Immune Regulation is taking an even bigger step toward advancing its goals.

Formerly known as Peptinnovate, the British biotech announced a $53.4 million Series B early Monday morning, helping to further advance two clinical programs in rheumatoid arthritis and asthma. Though those are the two initial indications the company is focusing on, CEO Jonathan Rigby told Endpoints News he hopes the candidates can be applied to a broad swath of autoimmune disorders.

Frank Zhang (AP Images)

Rocked by cus­toms in­ves­ti­ga­tion, Leg­end's CFO takes over as CEO Frank Zhang placed un­der house ar­rest

When Frank Zhang stepped down from GenScript — the contract research group he’s run for 18 years — to take up the CEO post at its CAR-T focused spinout Legend Biotech, he assured analysts that he was in for the long haul.

Just 49 days later, though, he’s been forced to hand back the title.

In a dramatic turn of events, Legend disclosed that Zhang is under house arrest in China as part of a customs investigation involving GenScript. While he remains the chairman, CFO Ying Huang has been tapped to double as interim CEO.

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Israel Lowy (Regeneron)

#ES­MO20: 'As good as any PD-1 out there': Re­gen­eron flash­es PD-(L)1 lung can­cer da­ta to ri­val Mer­ck

Regeneron entered the PD-(L)1 game late, so they devised a two-pronged strategy to catch up with Big Pharma rivals: They would push it into cancers where PD-1s had yet been tested, and they would prove that it’s as powerful in the big indications as any other on the market.

They cleared a hurdle on the first goal Friday, showing a 31% response in patients with the rare skin cancer basal cell carcinoma. And with the data they’re rolling out Monday, Regeneron cancer chief Israel Lowy is ready to declare success on the second.

Eli Lilly CSO Dan Skovronsky (file photo)

UP­DAT­ED: #ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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