Or­chard se­cures re­im­burse­ment for gene ther­a­py; UK fines drug­mak­ers $47M for an­ti­com­pet­i­tive scheme

Or­chard Ther­a­peu­tics an­nounced this week that the com­pa­ny has agreed to a re­im­burse­ment deal with Eng­land’s NHS for cov­er­age of its gene ther­a­py Lib­meldy.

Al­though Or­chard didn’t say how much NHS would pay for the ther­a­py, the deal is no­table be­cause of the dif­fi­cul­ties gene ther­a­py de­vel­op­ers have had com­ing to terms with pay­ers, even when the ther­a­pies have been ap­proved. In the most high-pro­file ex­am­ple, blue­bird bio pulled all of its gene ther­a­pies out of Eu­rope last year, say­ing coun­tries were not of­fer­ing a price com­men­su­rate with the val­ue of its drugs for two rare blood dis­or­ders and a rare neu­ro­log­i­cal con­di­tion.

Or­chard’s ther­a­py is a one-time treat­ment for metachro­mat­ic leukody­s­tro­phy, a pro­gres­sive and ul­ti­mate­ly fa­tal dis­or­der in which a type of fat builds up in the brain, ner­vous sys­tem and oth­er tis­sues. A Lancet pub­li­ca­tion last month showed that 26 out of 29 treat­ed pa­tients were still alive, had shown sig­nif­i­cant im­prove­ments in mo­tor func­tion com­pared to his­tor­i­cal con­trols and “most dis­played nor­mal cog­ni­tive de­vel­op­ment.”

UK fines drug­mak­ers over an­ti-com­pet­i­tive scheme

The UK’s Com­pe­ti­tion and Mar­kets Au­thor­i­ty fined four com­pa­nies a com­bined £35 mil­lion — $47 mil­lion — for a scheme al­leged­ly de­signed to sti­fle com­pe­ti­tion in the mar­ket for a com­mon an­ti-nau­sea drug.

The agency said Al­liance Phar­ma­ceu­ti­cals ap­point­ed a com­pa­ny called Fo­cus to dis­trib­ute the gener­ic drug prochlor­per­azine in the coun­try. Mean­while, they al­so paid two oth­er drug com­pa­nies, Lex­on and Medre­ich, a share of the prof­its in an ex­change for an agree­ment that the two would not al­so launch prochlor­per­azine in the same pe­ri­od.

As a re­sult, the CMA said, from 2013 to 2017, the price of prochlor­per­azine in­creased 700%. Be­tween 2014 and 2018, it said, the amount the NHS spent on the drug rose from £2.7 mil­lion to around £7.5 mil­lion, even though the amount used in the UK fell.

“The size of the fines re­flects the se­ri­ous­ness of this breach. These firms con­spired to sti­fle com­pe­ti­tion in the sup­ply of this im­por­tant med­ica­tion, so that the NHS – the main buy­er of the drugs – lost the op­por­tu­ni­ty for in­creased choice and low­er prices,” CMA chief An­drea Coscel­li said. “While the arrange­ment was in place, the price in­creased sig­nif­i­cant­ly for a drug that peo­ple re­ly on to man­age de­bil­i­tat­ing nau­sea, dizzi­ness and mi­graines.”

Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

Roger Perl­mut­ter lines up deals, fresh fund­ing at Eikon; Sec­ond RSV vac­cine ap­proved; Sev­er­al biotechs flash­ing red; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As you come back to our website this weekend for ASCO news, don’t forget to check out our updated event lineup at BIO, which will cover everything from the current state of VC investing in biotech to top pharma R&D chiefs discussing how to make pipeline decisions.

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Peter van de Sande, Synaffix CEO

Lon­za shells out $107M cash to snap up Synaf­fix and its ADC plat­form

After lining up a string of partnerships over the years, Dutch antibody-drug conjugate specialist Synaffix has found a new home: Lonza, the contract development and manufacturing giant.

Lonza is paying about $107 million (€100 million) in cash to acquire Synaffix, with up to $64 million (€60 million) in “additional performance-based consideration” on the table. Synaffix’s ADC tech platform will now become part of Lonza’s offering for biopharma clients, lending its bioconjugate technologies to not just ADCs but also targeted gene therapy, immune cell engagers and other applications.

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Full TIG­IT da­ta from Gilead, Ar­cus show low­er PFS rates than De­cem­ber read­out: #AS­CO23

CHICAGO — Gilead and Arcus unveiled a fuller snapshot of a Phase II study testing their experimental cancer immunotherapy combo that showed lower progression-free survival rates than its previous update, results that are likely to spark further debate over the closely-watched clinical trial.

Last December, the anti-TIGIT/anti-PD-L1 combo, positioned as a first-line treatment for non-small cell lung cancer, recorded data that drew mixed reactions. The latest analysis, presented Saturday afternoon at ASCO, included only a handful more patients than the previous update, but PFS rates fell — in one cohort by nearly three months.

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Bris­tol My­er­s' Op­di­vo keeps can­cer at bay in more lym­phoma pa­tients than Seagen's Ad­cetris in PhI­II: #AS­CO23

CHICAGO — In a study pitting Seagen’s Adcetris against Bristol Myers Squibb’s Opdivo in newly diagnosed patients with advanced classic Hodgkin lymphoma, a greater proportion of those who received Opdivo saw no cancer growth at one year compared to those who got Adcetris.

In addition, patients in the Opdivo arm of the Phase III trial reported reduced toxicities, according to lead investigator Alex Herrera, a hematologist-oncologist at City of Hope’s cancer cancer in Duarte, CA. Notably, the trial included more than 200 children across both arms. Generally, more than half of children with advanced Hodgkin lymphoma receive radiation therapy, but in this trial, dubbed SWOG S1826, only a handful of patients in the two arms received radiotherapy, sparing many children from long-term side effects of radiation.

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Servi­er’s vo­rasi­denib stalls pro­gres­sion of brain can­cer by 61% in piv­otal PhI­II IN­DI­GO study: #AS­CO23

An experimental pill from Servier Pharmaceuticals showed potentially practice-changing results in a narrow group of brain cancer patients, cutting the risk of their cancers progressing by 61%, according to a late-stage clinical trial.

The drug, vorasidenib, is a precision medicine that only works in certain people whose cancer carries mutations in one of two genes called IDH1/2. Doctors hope that the therapy will delay the need for chemotherapy or radiation, which are often used to combat relapses in patients who’ve previously undergone surgery to remove brain tumors.

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As­traZeneca tri­al shows mod­est ben­e­fit in ovar­i­an can­cer, but doc­tors say it's hard to ap­ply find­ings: #AS­CO23

CHICAGO — Adding AstraZeneca’s Imfinzi and Lynparza to the treatment regimen for patients with advanced ovarian cancer and no BRCA mutation extended progression-free survival (PFS) by five months, according to interim data released at the ASCO annual meeting Saturday morning.

However, the design of the Phase III study obscures how much Imfinzi is contributing to the PFS extension, doctors said, making it difficult to apply the findings to clinical practice.

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Keytru­da be­fore and af­ter lung can­cer surgery cuts re­lapse risk by 42%, but doesn’t im­prove sur­vival: #AS­CO23

CHICAGO — Merck has found partial success with its latest effort to more aggressively treat earlier stages of lung cancer.

On Saturday the pharma giant announced results from a large trial in which patients received Merck’s immunotherapy Keytruda plus chemotherapy before surgeons removed their tumors, followed by another course of Keytruda afterward.

The Phase III study, called KEYNOTE-671, enrolled 800 people with the early stages of the most common kind of lung cancer: non-small cell lung cancer, or NSCLC. Everyone got chemo before surgery, and half also got Keytruda before and after. At two years, 62.4% of those who got Keytruda kept their cancer at bay, compared to 40.6% who got a placebo.

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Grail’s blood test charts path for di­ag­nos­ing pa­tients sus­pect­ed of hav­ing can­cer in large study: #AS­CO23

Grail’s vision is simple but bold. The blood testing company has long held that people are often diagnosed with cancer too late. If seemingly healthy people were screened for early signs of the disease before symptoms appear, they may be able to get more effective treatments that nip cancer in the bud.

That premise is the basis of Grail’s commercial blood test, Galleri, which searches for the genetic fingerprints of cancer in the blood. The test, launched in 2021, reaped $55 million in sales last year, but now the company is setting its sights on a new market: patients suspected of having cancer due to symptoms such as abdominal pain, rectal bleeding or unexplained weight loss. Rather than administering expensive scans or conducting invasive biopsies right away, Grail hopes doctors will consider a simple blood test.

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