Four years af­ter EU ap­proval, NICE fi­nal­ly backs Revlim­id use in cer­tain first-line mul­ti­ple myelo­ma pa­tients

Four years af­ter the Eu­ro­pean Com­mis­sion sanc­tioned the use of Cel­gene’s flag­ship Revlim­id to treat adults with pre­vi­ous­ly-un­treat­ed mul­ti­ple myelo­ma who are not el­i­gi­ble for trans­plant, Eng­land’s Na­tion­al Health Ser­vice (NHS) will adopt its use as the first or sec­ond line of de­fense — in cer­tain pa­tients. Al­though the block­buster drug is rou­tine­ly used as a back­bone treat­ment in much of the de­vel­oped world, un­til now, the drug was on­ly cleared for third-line use by NICE.

Revlim­id, which gen­er­at­ed rough­ly $9.7 bil­lion in glob­al 2018 sales, has long been Cel­gene’s key­stone treat­ment, but its use in Eng­land had been re­strained due to pric­ing dis­agree­ments. The drug is al­so cru­cial to Bris­tol-My­er’s $BMY $74 bil­lion bet on the big biotech (al­though the loss of patent pro­tec­tion is loom­ing).

Mul­ti­ple myelo­ma is a life-threat­en­ing form of blood can­cer, which orig­i­nates in the bone mar­row, and af­fects rough­ly 17,500 peo­ple in the UK at any giv­en time, ac­cord­ing to char­i­ty Myelo­ma UK, which es­ti­mates each year that about 5,700 new pa­tients are di­ag­nosed with the dis­ease.

NICE pub­lished two pieces of fi­nal draft guid­ance on Fri­day. In the first re­port, NICE rec­om­mend­ed the use of Revlim­id — known chem­i­cal­ly as lenalido­mide — in com­bi­na­tion with the cor­ti­cos­teroid dex­am­etha­sone as an op­tion for treat­ment-naive adults with mul­ti­ple myelo­ma who are not el­i­gi­ble for a stem cell trans­plant and can­not take thalido­mide. This new op­tion could ben­e­fit about 2,100 pa­tients, NICE said.

Thalido­mide (or borte­zomib, or Take­da’s $TAK Vel­cade, for those who can’t take thalido­mide) re­mains the first line of de­fense, al­though NICE ac­knowl­edged that se­vere side-ef­fects as­so­ci­at­ed with its use can rule out its adop­tion. How­ev­er, “(l)enalido­mide plus dex­am­etha­sone can­not be rec­om­mend­ed for un­treat­ed mul­ti­ple myelo­ma in peo­ple who could take thalido­mide be­cause this would not be cost ef­fec­tive,” NICE un­der­scored.

Lenalido­mide is avail­able as a 21‑cap­sule pack, and its cost varies per dose, up to £4,368 for the high­est 25 mg dose/pack. But, Cel­gene $CELG has now agreed to a (con­fi­den­tial) dis­count that has sat­is­fied NICE. 

In the sec­ond re­port, NICE backed the use of lenalido­mide as an op­tion for treat­ing mul­ti­ple myelo­ma in adults if they have had on­ly one pre­vi­ous ther­a­py, in­clud­ing borte­zomib. Par­tic­u­lar­ly for those that have been first treat­ed with borte­zomib, the on­ly next treat­ment in the doc­tor’s tool­box is chemother­a­py — how­ev­er, ev­i­dence in­di­cates that the lenalido­mide+dex­am­etha­sone is more ef­fec­tive than cy­to­tox­ic chemother­a­py. In one clin­i­cal study, pa­tients tak­ing lenalido­mide lived on av­er­age 7 months longer, NICE said.

About 320 pa­tients are ex­pect­ed to ben­e­fit, it added.

The most ‘plau­si­ble’ cost-ef­fec­tive­ness es­ti­mate for lenalido­mide plus dex­am­etha­sone may be above the range that NICE nor­mal­ly con­sid­ers be­ing a cost-ef­fec­tive use of NHS re­sources, re­searchers high­light­ed in the re­port, but con­ced­ed that since lenalido­mide has been rec­om­mend­ed for use as a first treat­ment (for which it is cost-ef­fec­tive) — the need for lenalido­mide as a sec­ond treat­ment will like­ly de­crease be­cause peo­ple are more like­ly to have it as a first treat­ment in the fu­ture.

Last month, NICE al­so backed the use of J&J’s $JNJ Darza­lex in com­bi­na­tion with Vel­cade as a sec­ond-line treat­ment for mul­ti­ple myelo­ma via the can­cer drugs fund.

Cel­gene, mean­while, is ad­vanc­ing the use of triplet mul­ti­ple myelo­ma ther­a­pies. On Thurs­day, the EU ap­proved 1) Revlim­id in com­bi­na­tion with Vel­cade and dex­am­etha­sone for adult pa­tients with pre­vi­ous­ly un­treat­ed mul­ti­ple myelo­ma who are not el­i­gi­ble for trans­plant; 2) Im­novid in com­bi­na­tion with Vel­cade and dex­am­etha­sone in adult pa­tients with mul­ti­ple myelo­ma, who have re­ceived at least one pri­or treat­ment reg­i­men in­clud­ing Revlim­id.


Im­age Source: As­so­ci­at­ed Press

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.


ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology


ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development


CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

SEC calls out lit­tle Ther­a­peu­tic­sMD for its in­sid­er con­tacts with an­a­lysts to boost share price, then halt rout

Back in May 2017, following an FDA rejection, TherapeuticsMD saw its share price plummet to the lowest levels in two years. The little Florida biotech eventually found its way back to the good side of regulators, scoring a curious OK a year later for its therapy preventing vaginal pain during sex. But the SEC is now accusing it of selectively disclosing nonpublic information in attempts to manipulate its stock.

In two instances in June and July of 2017, TherapeuticsMD allegedly violated the Regulation Fair Disclosure rule by sharing material information with certain sell-side analysts and not the public, resulting in a more favorable stock move than otherwise would be expected.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Therapists Marcela Ot'alora and Bruce Poulter are trained to conduct MDMA-assisted psychotherapy. In this reenactment, they demonstrate how they help guide and watch over a patient who is revisiting traumatic memories while under the influence of MDMA. (Photo: Multidisciplinary Association for Psychedelic Studies)

MD­MA, now in Phase III, shows promise as a PTSD treat­ment

The first time Lori Tipton tried MDMA, she was skeptical it would make a difference.

“I really was, at the beginning, very nervous,” Tipton said.

MDMA is the main ingredient in the club drug known as ecstasy or molly. But Tipton wasn’t taking pills sold on the street to get high. She was trying to treat her post-traumatic stress disorder by participating in a clinical trial.

After taking a dose of pure MDMA, Tipton lay in a quiet room with two specially trained psychotherapists. They sat next to her as she recalled some of her deepest traumas, such as discovering her mother’s body after Tipton’s mother killed two people and then herself in a murder-suicide.

Ted Ashburn. Oncorus

Cowen, Per­cep­tive lead $79.5M Se­ries B for 's­tand­out' biotech shep­herd­ing on­colyt­ic virus to clin­ic

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin Bio Ventures and UBS Oncology Impact Fund in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

Fu­til­i­ty analy­sis au­gurs de­feat in piv­otal tri­al test­ing of Nu­Cana's lead drug in metasta­t­ic pan­cre­at­ic can­cer

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.