No­var­tis an­nounces pos­i­tive da­ta from halt­ed Zol­gens­ma tri­al

The tri­al may be on hold, but No­var­tis says the drug works.

No­var­tis an­nounced to­day new da­ta on a de­liv­ery route to make the gene ther­a­py Zol­gens­ma ap­plic­a­ble and ac­ces­si­ble to old­er kids with spinal mus­cu­lar at­ro­phy, even as the Phase I/II tri­al on that route re­mains on hold. The FDA paused the tri­al in Oc­to­ber af­ter pre­clin­i­cal stud­ies showed loss of neur­al func­tion in some mice. The hold came af­ter an in­dus­try-rock­ing scan­dal over how No­var­tis han­dled da­ta-tam­per­ing in a dif­fer­ent set of pre­clin­i­cal Zol­gens­ma stud­ies.

No­var­tis sub­sidiary AveX­is, which de­vel­oped and mar­kets Zol­gens­ma, said that in the me­di­an dos­ing arm of their STRONG tri­al, the 2-year-old to 5-year-old pa­tients met the pri­ma­ry end­point: They im­proved an av­er­age of 6.0 points on the Ham­mer­smith Func­tion­al Mo­tor Scale-Ex­pand­ed, a met­ric used to mea­sure mus­cle func­tion in kids with spinal mus­cu­lar at­ro­phy. AveX­is said that was twice the im­prove­ment re­quired to be clin­i­cal­ly mean­ing­ful and re­flect­ed ad­vances in three to six mo­tor skills.

David Lennon No­var­tis

In a press re­lease, AveX­is CEO David Lennon praised the re­sults and hint­ed he hoped they would con­vince reg­u­la­tors to re­sume the tri­al. He used the ex­per­i­men­tal com­pa­ny name of the drug.

“STRONG da­ta demon­strat­ed po­ten­tial best-in-cat­e­go­ry pro­file with re­mark­able mo­tor func­tion im­prove­ment fol­low­ing a sin­gle, one-time in­trathe­cal dose,” Lennon said. “We look for­ward to shar­ing these da­ta with reg­u­la­tors to fur­ther our dis­cus­sions to­ward reg­is­tra­tion of in­trathe­cal AVXS-101.”

Ap­proved last year, Zol­gens­ma was the sec­ond gene ther­a­py to reach the mar­ket in the US and just the sec­ond-ever treat­ment for spinal mus­cu­lar at­ro­phy af­ter the 2016 ap­proval of Io­n­is’s and Bio­gen’s Spin­raza. The ther­a­py was lim­it­ed, though, to pa­tients un­der the age of 2. The swift­ness of the ge­net­ic dis­or­ders re­duced the odds of suc­cess the old­er pa­tients got. Most kids with SMA type 1 — the first ap­proved in­di­ca­tion — do not live past the age of 2.

Peo­ple with SMA type 2, how­ev­er, can live in­to ear­ly adult­hood, and No­var­tis has been try­ing to test if an in­trathe­cal in­jec­tion of  Zol­gens­ma — as op­posed to an in­tra­venous one — can im­prove their prog­no­sis.

The hold on the tri­al af­fect­ed the high­est dos­ing arm of the study. Since Oc­to­ber, the Swiss gi­ant has tried to get the FDA to lift the halt, point­ing to the fact that the neur­al dam­age shown in the pre­clin­i­cal study has nev­er ap­peared across the 300-plus pa­tients who have re­ceived the gene ther­a­py. The com­pa­ny said they ex­pect a de­ci­sion from the FDA next quar­ter.

A small, ob­scure biotech just won big with their IPO. In this mar­ket. Are you kid­ding me?

How could a small, largely unknown biotech that emerged from stealth mode just months ago with early-stage cancer programs jump onto Wall Street in the middle of a Category 6 financial hurricane and sail through with a $165 million IPO?

And what does that mean for the rest of the industry waiting to see just how much damage global lockdowns will wreak on clinical development?

The biotech is a company called Zentalis. The crew there nabbed an $85 million crossover round late last year — notably waiting 5 years before waving the numbers around to attract attention, according to my read of a FierceBiotech story. Perceptive joined in, but the syndicate was not in general the kind of marquee affair that gets tongues wagging.

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Bob Nelsen at the Milken Institute Global Conference on April 29, 2019 in Beverly Hills, California. (Photo by Michael Kovac/Getty Images)

ARCH chief Bob Nelsen has $1.5B to prove 2 sim­ple points: ‘We’re in the most in­no­v­a­tive time ever’ and in­vestors are stay­ing

ARCH co-founder and managing director Bob Nelsen has a well known yen for the home run swing, betting big on potentially transformative meds and tech and the biotech teams he helps bring together. He thrives and bleeds on the cutting edge. And now Nelsen and the ARCH group have debuted 2 big funds to prove that this is the time for the best of biotech to shine — deadly pandemic be damned.

Two new funds, ARCH Venture Fund X and ARCH Venture Fund X Overage, gathered a combined $1.46 billion. And that’s a record. ARCH Venture Fund IX and ARCH Venture Fund IX Overage closed in 2016 with a combined $1.1 billion. ARCH Venture Fund VIII and ARCH Venture Fund VIII Overage closed in 2014 with a combined $560 million.

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Lil­ly Asia Ven­tures co-leads $100M+ round for Chi­nese biotech and its late-stage lu­pus drug

Can a Chinese biotech deliver the first new lupus drug in decades? A high-profile group of VCs are betting on it.

Lilly Asia Ventures and Lake Bleu Capital are the co-headliners for RemeGen’s latest raise, which brought in more than $100 million. Hudson Bay Capital and Vivo Capital — which, like LAV, also invested in a pre-IPO round for Legend Biotech unveiled today — chimed in, as did Janchor Partners and OrbiMed.

GSK's asth­ma bi­o­log­ic Nu­cala is one step clos­er to ap­proval in key chron­ic rhi­nos­i­nusi­tis pop­u­la­tion

Months after GSK’s Nucala cleared in a pivotal rare blood disorder study, the asthma biologic has scored in a late-stage trial in chronic rhinosinusitis patients with nasal polyps.

The British drugmaker on Friday disclosed data from the SYNAPSE study, which tested Nucala (also known as mepolizumab) against a placebo on top of standard-of-care in more than 400 patients, all of whom had a history of previous surgery (approximately one in three had ≥3 surgeries) and required surgery due to severe symptoms and bigger polyps.

Servi­er bags an an­ti­body spe­cial­ist in its lat­est on­col­o­gy M&A deal with plans to add the plat­form tech

Whatever Servier learned about Symphogen during their 2-year development alliance must have significantly whetted their appetite for an acquisition.

Paris-based Servier announced Friday that it has struck a deal to buy out the antibody expert. The acquisition comes 2 years after Servier acquired Shire’s cancer business for $2.4 billion. They’ve been working with Symphogen on a slate of programs, including some favs – PD-1, LAG3 and TIM3 — where they are looking to differentiate themselves from the more prominent drugs in these niches.

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GSK vac­cine chief heads for AIDS vac­cine ini­tia­tive; Pfiz­er en­lists Sue Desmond-Hell­mann to its board of di­rec­tors

→ Rip Ballou, who until very recently led vaccine research and development at GlaxoSmithKline, is joining the International AIDS Vaccine Initiative (IAVI) to lead its USAID-funded ADVANCE program. The program uses a network of researchers and institutions in Africa to help develop a vaccine for HIV. Ballou had worked at GSK since 2010 and has led global vaccine R&D since 2015. Prior to that he held posts at the Bill & Melinda Gates Foundation, a different post at GSK, Medimmune, and Walter Reed Army Institute of Research.  IAVI is led by Mark Feinberg, the former CSO of Merck Vaccines. 

Aaron Royston, venBio

In­vest­ing in the time of coro­n­avirus: the good, the bad and the hope­ful, as biotech VC firms close funds worth $3B

Apart from disrupting biopharma R&D and regulatory timelines, the coronavirus pandemic has inevitably ravaged financial markets and eroded investor risk appetite. Investing in the time of coronavirus feels reckless, but if biotech venture funds are any indication, the time is ripe.

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Ahead of US IPO, Leg­end Biotech adds $150M, top-tier in­vestors to back CAR-T pipeline

Last month Nanjing Legend Biotech revealed that it sees, and was quietly planning for, a future as a public company in the US, separate but still tied to its former parent, Chinese CRO GenScript. It’s evidently a vision that enticed investors, drawing marquee names for a pre-IPO round.

The Series A fetched a whopping $150.5 million from Hudson Bay Capital Management, Lilly Asia Ventures, Vivo Capital, RA Capital Management and JJDC, the venture arm of J&J. The pharma giant has helped fund Legend’s CAR-T work with the $350 million upfront payment it handed over to partner on the lead BCMA program.

Drug dis­cov­ery in the age of coro­n­avirus

Developing new drugs is incredibly hard. That’s why, despite superhuman efforts from the industry, we’re still looking at 12-18 months minimum before we can realistically hope for a vaccine for Covid-19, and probably months before there’s a proven viable drug treatment.

But our increasing ability to begin to industrialize the drug discovery and development process through an engineering approach means that we have more hope for speeding up this process than ever before — and not just to defeat coronavirus, but to benefit the development of all new medicines in the future.