Am­i­cus, Penn ex­pand gene ther­a­py re­search agree­ment in­to five-year col­lab­o­ra­tion

Apart from get­ting the FDA to have a change of heart on its Fab­ry dis­ease ther­a­py, Galafold, last year, Am­i­cus Ther­a­peu­tics joined forces with the Uni­ver­si­ty of Penn­syl­va­nia to work on gene ther­a­pies for four dif­fer­ent dis­or­ders — Pompe dis­ease, Fab­ry dis­ease, CD­KL5 de­fi­cien­cy and an undis­closed rare meta­bol­ic dis­or­der. On Wednes­day, the two ex­pand­ed their col­lab­o­ra­tion — to in­clude ad­di­tion­al lyso­so­mal dis­or­ders and rare dis­eases.

John Crow­ley Am­i­cus Ther­a­peu­tics

The part­ner­ship was inked to bring to­geth­er Penn’s AAV vec­tor tech­nol­o­gy (AAVs are small, non-path­o­gen­ic virus­es with a genome of sin­gle-strand­ed DNA, which can in­sert ge­net­ic ma­te­r­i­al at a spe­cif­ic site), which is en­gi­neered to en­hance tar­get­ing, safe­ty and gene de­liv­ery — with Am­i­cus’ $FOLD tech­nol­o­gy de­signed to op­ti­mize pro­tein ex­pres­sion, se­cre­tion, and the up­take of the tar­get pro­tein.

Un­der the new five-year agree­ment, the two en­ti­ties will al­so work to­geth­er on gene ther­a­pies for Nie­mann-Pick Type C (NPC) and two forms of San­fil­ip­po Syn­drome. In ad­di­tion, Am­i­cus has the op­por­tu­ni­ty to col­lab­o­rate with Penn’s Gene Ther­a­py Pro­gram (GTP) to de­vel­op pro­grams for the ma­jor­i­ty of lyso­so­mal dis­or­ders and 12 ad­di­tion­al rare dis­eases, in­clud­ing Rett Syn­drome, An­gel­man Syn­drome, My­oton­ic Dy­s­tro­phy and se­lect oth­er mus­cu­lar dy­s­tro­phies. Am­i­cus will in­ject $10 mil­lion an­nu­al­ly for five years in­to GTP’s dis­cov­ery re­search pro­gram — with the abil­i­ty to ex­tend.

James Wil­son GTP

Am­i­cus’ ex­per­i­men­tal drug for Pompe dis­ease (an in­her­it­ed lyso­so­mal stor­age dis­or­der), AT-GAA, did not gen­er­ate much en­thu­si­asm for ac­cel­er­at­ed re­view by the FDA, de­spite CEO John Crow­ley’s best ef­forts last Sep­tem­ber.

The com­pa­ny’s work with Penn on Pompe dis­ease, mean­while, has gen­er­at­ed pos­i­tive da­ta.

“We have al­ready seen high­ly en­cour­ag­ing pre­clin­i­cal re­sults and proof-of-con­cept in Pompe dis­ease through our ex­ist­ing col­lab­o­ra­tion…and I be­lieve that we can fur­ther ex­pand and ac­cel­er­ate our ef­forts to rapid­ly de­vel­op gene ther­a­pies for many more pa­tients,” said James Wil­son, pro­fes­sor of med­i­cine and pe­di­atrics at Penn’s Perel­man School of Med­i­cine, in a state­ment.

Im­age: Shut­ter­stock

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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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 with any issues.

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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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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 and UBS 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.

UP­DAT­ED: Pay­back? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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FDA de­ci­sion on Ver­tex's CF triple will come just ahead of planned CEO shake­up

Vertex has clinched a priority review for the all-important cystic fibrosis triple that will blaze the trail for treating a large group of patients unhelped by its current drugs.

FDA regulators have set a PDUFA date of March 19, 2020, just a year after the Boston biotech posted positive Phase III results showing that people with two F508del mutations experienced statistically significant improvements in lung function after a 4-week regimen of VX-445, tezacaftor and ivacaftor. After reviewing 24-week data among patients with one F508del mutation and one minimal function mutation — and thoroughly comparing the VX-445 triple with another combo featuring VX-659 on scores like safety, drug-drug interactions, and photosensitivity — Vertex ultimately went with VX-445.