Capri­cor's DMD clin­i­cal tri­al meets pri­ma­ry end­points; Delaware court rules against Take­da in Lupron feud

Bev­er­ly Hills, CA-based biotech Capri­cor Ther­a­peu­tics re­vealed to­day fi­nal da­ta from its clin­i­cal tri­al eval­u­at­ing stem cell ther­a­py CAP-1002 for pa­tients in ad­vanced stages of Duchenne mus­cu­lar dy­s­tro­phy.

Duchenne is a rare mus­cle-wast­ing dis­ease caused by the ab­sence of dy­s­trophin, a pro­tein that helps keep mus­cle cells in­tact. It dis­pro­por­tion­ate­ly af­fects boys — and as of last year, af­fects rough­ly 6,000 in the Unit­ed States.

Ac­cord­ing to a Capri­cor state­ment, the HOPE-2 clin­i­cal tri­al met its pri­ma­ry ef­fi­ca­cy end­point of up­per limb per­for­mance, as well as var­i­ous skele­tal and car­diac end­points sug­gest­ing slow­ing of dis­ease pro­gres­sion.

Lin­da Mar­bán

The ran­dom­ized, dou­ble-blind­ed, place­bo-con­trolled Phase II clin­i­cal tri­al of CAP-1002 was con­duct­ed at nine lo­ca­tions across the US with no se­ri­ous safe­ty sig­nals iden­ti­fied, with the ex­cep­tion of two hy­per­sen­si­tiv­i­ty re­ac­tions ear­ly on.

“Now that we have clar­i­ty from the FDA and based on the strength of this da­ta set, we are poised to em­bark on the HOPE-3 piv­otal tri­al once we have se­cured an ap­pro­pri­ate part­ner that can help dri­ve CAP-1002 for­ward to­wards com­mer­cial­iza­tion,” said Capri­cor CEO Lin­da Mar­bán in a state­ment.

Eq­ui­ty court rules Take­da breached con­tract with Ab­b­Vie on prostate can­cer drug

A Delaware judge has is­sued a sep­a­rate rul­ing in fa­vor of Ab­b­Vie in the phar­ma gi­ant’s spat with Take­da, two weeks af­ter sid­ing with its Lupron part­ner.

In the lat­est rul­ing, the judge found Take­da guilty of breach of con­tract for fail­ing to sup­ply Ab­b­Vie with the prostate can­cer drug un­der their pri­or deal. Two weeks ago, the judge ruled that the Delaware Court of Chancery, where the case was heard, didn’t have the ju­ris­dic­tion to or­der Take­da to live up to its sup­ply agree­ment.

Ab­b­Vie had claimed that Take­da is re­spon­si­ble for sup­ply chain prob­lems that have led to world­wide short­ages of Lupron, a hor­mone drug that has been used to treat prostate can­cer. While Ab­b­Vie both de­vel­oped the med­i­cine and dis­trib­utes it in the US and Cana­da, Take­da is the drug’s on­ly man­u­fac­tur­er, sup­ply­ing it from its Hikari, Japan plant.

Lupron has been in short sup­ply world­wide since the com­pa­ny and the FDA dis­cov­ered “sig­nif­i­cant vi­o­la­tions” at the plant in No­vem­ber 2019. The judge told the com­pa­nies that an­oth­er tri­al is need­ed to de­ter­mine the ex­tent of the dam­ages caused by Take­da.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'


Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

FDA ad­comm to de­cide on mol­nupi­ravir EUA; Can­cer at­las un­veils new po­ten­tial drug tar­get

The FDA has another adcomm coming down the pipeline — this time on Covid-19 oral antiviral molnupiravir.

The federal agency’s advisory committee will meet on November 30th to go over Merck and Ridgeback’s EUA request for their investigational antiviral drug, and discuss the available data supporting its use in Covid-19 patients.

This comes two weeks after Merck claimed that their antiviral pill reduced the chance that newly diagnosed Covid-19 patients would be hospitalized or die by 50%. The pharma made the announcement after interim data on 775 patients in their clinical trial showed the antiviral’s potential.

FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.

Rahul Singhvi, Resilience CEO

A Bob Nelsen start­up turns to Har­vard to help sharp­en its tech, in­spir­ing first spin­out

One of Bob Nelsen’s latest projects is headed to Harvard.

Resilience, a company started with the goal of establishing itself as a “one-stop-shop” for companies looking to scale manufacturing, including for hard-to-develop cell and gene therapies, is less than a year old. Friday, it announced a five-year R&D deal with Harvard University that includes $30 million to develop biologics, including vaccines, nucleic acids and cell and gene therapies.