Roche/PTC edge clos­er to dis­rupt­ing mar­ket for SMA ri­vals Bio­gen and No­var­tis with more pos­i­tive da­ta

Roche and part­ner PTC Ther­a­peu­tics have bro­ken out de­tailed pos­i­tive da­ta back­ing their spinal mus­cu­lar at­ro­phy ther­a­py, which is ex­pect­ed to have a good shot at eclips­ing Spin­raza’s mar­ket lead­er­ship.

The FDA is set to make its de­ci­sion on the ther­a­py, ris­diplam, by May 24. If ap­proved — the drug is ex­pect­ed to gen­er­ate be­tween $2 bil­lion and $3 bil­lion in peak sales — and com­pete with Bio­gen’s first-to-mar­ket Spin­raza and No­var­tis’ most-ex­pen­sive-drug-in-the-world Zol­gens­ma.

On Thurs­day, the com­pa­nies re­port­ed de­tailed da­ta from the SUN­FISH tri­al, which test­ed ris­diplam against a place­bo in 180 pa­tients aged 2-25 years with type 2 and 3 SMA, a rare ge­net­ic mus­cle-wast­ing dis­ease. Most pa­tients in the tri­al were old­er, and a rel­a­tive­ly more se­vere form of the dis­ease. The main goal of the study — which was met — was the change in mo­tor func­tion af­ter one year. Roche ini­tial­ly an­nounced the pri­ma­ry end­point had been met last No­vem­ber.

The to­tal mean change in pa­tients re­ceiv­ing ris­diplam was sig­nif­i­cant­ly high­er than place­bo (1.55 point mean dif­fer­ence; p=0.0156). As ex­pect­ed, the strongest re­spons­es ver­sus place­bo were ob­served in the youngest pa­tients aged be­tween 2 and 5 years (78.1% ver­sus 52.9% achiev­ing ≥3 point in­crease).

It is hard to com­pute the clin­i­cal mean­ing­ful­ness of ris­diplam based on ab­solute 1.55 mean point re­duc­tion, even though sta­tis­ti­cal sig­nif­i­cance was hit, Sun­Trust Robin­son Humphrey an­a­lyst Robyn Kar­nauskas wrote in a note.

PTC man­age­ment has pre­vi­ous­ly sug­gest­ed that a 2-point dif­fer­ence would be clin­i­cal­ly mean­ing­ful us­ing the Mo­tor Func­tion Mea­sure scale (MFM-32) scale, but since the mean point dif­fer­ence was 1.55, clin­i­cal mean­ing­ful­ness may not be so ev­i­dent at first blush, she said.

These re­sults sup­ple­ment pos­i­tive da­ta from 41 pa­tient-FIRE­FISH study, which test­ed ris­diplam in pa­tients aged 1-7 months with type 1 SMA.

Ris­diplam is strate­gi­cal­ly po­si­tioned to have strong up­take in the type 2/3 SMA pop­u­la­tion, Cred­it Su­isse’s Mar­tin Auster wrote in a re­cent note. “While we ex­pect Zol­gens­ma to cap­ture the vast ma­jor­i­ty of the in­ci­dence Type 1/2 SMA pop­u­la­tion, we an­tic­i­pate ris­diplam will emerge as the dom­i­nant ther­a­py for old­er SMA pa­tients.”

Bio­gen se­cured the first-ever SMA ap­proval and has since been dosed in over 9,300 pa­tients. How­ev­er, com­par­ing Bio­gen’s da­ta to the Roche/PTC re­sults in SMA type 2/3 pa­tients is dif­fi­cult giv­en the pri­ma­ry end­points are dif­fer­ent in the two tri­als, in ad­di­tion to dif­fer­ent en­roll­ment cri­te­ria.

“While the im­pact of No­var­tis’ Zol­gens­ma has been rel­a­tive­ly mut­ed, we think that the like­ly ap­proval and launch of ris­diplam could im­pact Bio­gen’s Spin­raza fran­chise,” Cred­it Su­isse’s Evan Seiger­man wrote in a re­cent note. “We al­so think that Roche could price ris­diplam be­low Spin­raza to help en­cour­age use – es­pe­cial­ly in T2 or T3 pa­tients.”

Spin­raza, an an­ti­sense oligonu­cleotide, is in­ject­ed in the spine every four months fol­low­ing ini­tial load­ing dos­es. Zol­gens­ma, a gene-ther­a­py, is de­signed to be a one-shot cure, while ris­diplam is a dai­ly oral treat­ment, en­gi­neered to work by tweak­ing how the SMN2 gene is spliced, which rais­es func­tion­al SMN pro­tein lev­els in both the cen­tral ner­vous sys­tem and pe­riph­er­al tis­sues.

SMA is rare, af­fect­ing 1 per 8,000 to 10,000 peo­ple glob­al­ly, but rep­re­sents a lu­cra­tive bat­tle­ground for these drug­mak­ers. Spin­raza, launched in late 2016, car­ries a list price of $750,000 for the first year and $375,000 an­nu­al­ly there­after. Zol­gens­ma — on­ly ap­proved for pa­tients un­der the age of 2 — caused stick­er shock with its $2.1 mil­lion price tag and the in­evitable push­back from pay­ers, al­though No­var­tis has em­pha­sized that its five-year in­stall­ment plan and cu­ra­tive po­ten­tial makes it worth it.

“While there are al­ready 2 ef­fi­ca­cious prod­ucts ap­proved for SMA in the US, (Roche) man­age­ment…see a very large op­por­tu­ni­ty giv­en the con­ve­nient oral for­mu­la­tion of ris­diplam, num­ber of pa­tients not el­i­gi­ble for gene ther­a­py, and small up­front price com­pared to gene ther­a­py and oligonu­cleotide com­peti­tors,” SVB Leerink an­a­lysts wrote in a re­cent note.

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.

Mi­rati’s drug sitra­va­tinib flops PhI­II in com­bo with Op­di­vo for cer­tain lung can­cer

Mirati Therapeutics’ path to a second drug approval will likely have to wait. The San Diego biotech company said Wednesday that its investigational lung cancer drug failed a Phase III trial testing it in combination with Bristol Myers Squibb’s Opdivo.

The drug, sitravatinib, and Opdivo weren’t better than the chemo drug docetaxel at keeping patients alive, Mirati said in a press release. The spectrum-selective kinase inhibitor missed the primary goal of overall survival in patients with second- or third-line advanced non-squamous, non-small cell lung cancer.

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End­points 20(+2) un­der 40, 2023; Bio­phar­ma's high­est-paid CEOs; N-of-1 CRISPR sto­ry goes on af­ter tragedy; 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.

We will be off Monday in observance of Memorial Day — and when we get back, it will be a straight march to ASCO, BIO and more. Enjoy the (long) weekend!

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Rich Horgan (R) with his late brother, Terry

Rich Hor­gan spear­head­ed a gene ther­a­py for his broth­er. The tri­al end­ed in tragedy, but the work con­tin­ues for more pa­tients

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

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Bio­phar­ma's 20 high­est-paid CEOs of 2022, each bring­ing in $20M+ pay­days

Even in a down year for much of the biopharma market, 20 CEOs brought in pay packages valued at more than $20 million, an Endpoints News analysis found.

Endpoints collected data on more than 350 CEO compensation packages, covering a wide range of pharma, biotech, and life sciences companies. All told, the 20 largest earners made over $725 million in 2022 — an average package of $36.4 million. Three brought in paydays over $50 million, and one CEO broke the $100 million mark.

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The 20(+2) un­der 40: Your guide to the next gen­er­a­tion of biotech lead­ers

This year’s list of 20 biotech leaders under the age of 40 includes a huge range of ambitions. Some of our honorees are planning to create the next big drug giant. Others are pushing the bounds of AI. One is working to revolutionize TB testing. All are compelling talents who are still young in age, but already far along in achievement.

And, as in years past, we went over. The 20 are actually 22 because of two double profiles that reflect how important teamwork is in the industry. As one of our honorees, Joe Illingworth of DJS Antibodies, told me in our interview, “It takes a village to raise a biotech.”

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Teresa Bitetti, Takeda's president of the global oncology business unit

Take­da wins pri­or­i­ty re­view for $400M col­orec­tal can­cer drug, li­censed from Hutchmed in Jan­u­ary

Takeda and Hutchmed scored a priority review Thursday afternoon for a colorectal cancer drug, the companies announced.

The experimental drug in question is fruquintinib, previously approved in China in 2018 to treat metastatic colorectal cancer. Takeda and Hutchmed are aiming to bring fruquintinib to the US and other countries outside China in the same indication, and the FDA set its decision date for Nov. 30 of this year.

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David Ricks, Eli Lilly CEO (Carolyn Kaster/AP Images)

Lil­ly gears up trio of PhI­II tri­als for its oral GLP-1 amid No­vo Nordisk, Pfiz­er com­pe­ti­tion

As Novo Nordisk and Pfizer disclose some data on their oral weight loss drugs in Phase III and II, respectively, Eli Lilly is beefing up its stance in the obesity field with three late-stage clinical trials of its next-generation GLP-1 agonist orforglipron.

The moves, disclosed in updates to the federal clinical trials database this week, put the Indianapolis drugmaker ahead of Pfizer, whose science chief has said the company will “cherry-pick” which of its mid-stage candidates to take deeper into the clinic after data late this year or early next.

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Douglas Love, Annexon CEO

An­nex­on’s GA drug miss­es on pri­ma­ry goal but win on vi­su­al acu­ity will be fo­cus of planned late-stage tri­al

Annexon’s complement inhibitor didn’t prove better than sham at reducing lesion growth in a leading cause of blindness, but the biotech still plans to move forward on the back of secondary endpoints showing visual acuity preservation, which will “certainly” be the primary goal in a late-stage trial to be discussed shortly with the FDA, CEO Douglas Love told Endpoints News. 

The California biotech’s ANX007 was not statistically significant compared to pooled sham, the comparator, at 12 months in patients with geographic atrophy, per a Wednesday presentation. In every-month dosing, the GA lesion area changed about 6.2% from baseline (p=0.526) and 1.3% (p=0.896) in the every-other-month group. In a March note, Jefferies analyst Suji Jeong said a reduction of 20% to 30% would be “encouraging.”

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