Roche boosts its gene ther­a­py game, ex­pand­ing part­ner­ship with 4D Mol­e­c­u­lar Ther­a­peu­tics

Join­ing the Big Phar­ma rush to pick up po­ten­tial­ly valu­able gene ther­a­py tech, Roche has ex­pand­ed its part­ner­ship with 4D Mol­e­c­u­lar Ther­a­peu­tics to work on a drug for night blind­ness, among oth­er oph­thal­mol­o­gy pro­grams.

David Kirn

Roche had first part­nered with the Emeryville, Cal­i­for­nia-based biotech back in 2016, when the duo was re­search­ing a small hand­ful of rare reti­nal dis­ease in­di­ca­tions. Now, it’s ex­pand­ed that com­mit­ment to take in oph­thal­mol­o­gy pro­grams that in­clude prospects for much larg­er mar­kets.

The progress of Spark and its gene ther­a­py hit Lux­tur­na — along with new part­ner­ships forged by its phar­ma brethren Pfiz­er and No­var­tis — like­ly laid the ground­work for Roche to re­con­sid­er its part­ner­ship with 4DMT.

Roche and 4DMT’s first col­lab­o­ra­tion pro­gram is for 4D-110, a clin­i­cal can­di­date to treat choroi­deremia (an in­her­it­ed form of night blind­ness that can show up in ear­ly child­hood). 4DMT is just now do­ing IND-en­abling stud­ies on the pro­gram. 4D-110 al­ready has some ad­vanced com­pe­ti­tion in the field, as Lon­don’s Night­star $NITE is al­so tack­ling this dis­ease with a Phase III gene ther­a­py drug.

4DMT’s edge would be its ade­no-as­so­ci­at­ed virus plat­form, which the com­pa­ny says is su­pe­ri­or to its ri­vals.

The part­ner­ship al­so in­cludes ad­di­tion­al clin­i­cal can­di­date pro­grams that are un­der­way to treat reti­nal dis­eases with high un­met need, 4DMT says. The com­pa­ny’s CEO David Kirn said this in a state­ment:

To­geth­er we elect­ed to ex­pand our part­ner­ship af­ter 4DMT com­plet­ed pro­pri­etary in­trav­it­re­al vec­tor dis­cov­ery and char­ac­ter­i­za­tion. We have cre­at­ed clear syn­er­gies be­tween 4D’s vec­tor dis­cov­ery, gene ther­a­py de­vel­op­ment and man­u­fac­tur­ing ca­pa­bil­i­ties and Roche’s ex­per­tise in late-stage clin­i­cal de­vel­op­ment and glob­al bi­o­log­ics com­mer­cial­iza­tion. The de­ci­sion to ex­pand our part­ner­ship rep­re­sents val­i­da­tion of our oph­thal­mol­o­gy plat­form, clin­i­cal can­di­dates and team.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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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.

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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.

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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.

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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.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.