Patrick Soon-Shiong (AP IMAGES)

Four years af­ter 'moon­shot' launch, Patrick Soon-Sh­iong has a big re­veal: a sin­gle pa­tient

Biotechs don’t typically unveil clinical data at the JP Morgan conference; it’s a gathering reserved for both the celebration and construction of deals, after all. But for Patrick Soon-Shiong, NantKwest chairman and CEO, it’s befitting.

Soon-Shiong, the inventor of Abraxane and controversial biotech billionaire, took the podium at JPM 2016 to launch a four-year moonshot to “subdue cancer by the start of the next decade.”

Now that 2020 is here, NantKwest is putting forth some new evidence for what it’s calling the “cancer breakthrough 2020”: one complete response in the expanded access portion of a Phase I trial.

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Catalent CEO John Chiminski speaks at an Endpoints News event at the JP Morgan conference in San Francisco, January 2020 [Jeff Rumans, Endpoints News]

Catal­ent chief John Chimin­s­ki trig­gers a $315M-plus wa­ger on the boom­ing cell ther­a­py mar­ket now in the mak­ing

Last year Catalent CEO John Chiminski invested more than a billion dollars to expand the contract manufacturer ahead of an explosion of gene therapy trials and expected approvals. He’s starting this year with a $315 million wager on the booming cell therapy field.

Just ahead of the market open Monday, Chiminski $CTLT laid out to me the details of his decision to buy a transatlantic player that has been building up manufacturing capability in CAR-Ts, TCRs, TILs (tumor-infiltrating lymphocytes) and mesenchymal stem cell therapies.

In exchange for the $315 million in cash, he’s getting MaSTherCell Global and its 32,000 square-foot facility in Houston and a 25,000 square-foot facility in Gosselies, Belgium. The manufacturing operation is in the process of building out a 60,000-square-foot addition in Belgium set to go live next year, expanding beyond clinical supplies to the commercial realm.

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The pow­er of prece­dent: How a one-off de­ci­sion by Janet Wood­cock es­tab­lished an un­in­tend­ed fast path­way at the FDA — what will fol­low?

When CDER chief Janet Woodcock overruled a group of senior regulators at the FDA a little more than 3 years ago to allow Sarepta to start marketing eteplirsen for Duchenne muscular dystrophy, she reassured some of her colleagues at the agency that it was the only time the biotech would win an approval based on a slight elevation of dystrophin production — a so-called “surrogate endpoint” used to help guide their accelerated approval.

The next time Sarepta arrived with a Duchenne drug — she said in several meetings about eteplirsen at the FDA — the biotech would be required to proffer data from a clinical trial with a hard endpoint, according to a regulatory expert with a close understanding of the internal workings of the agency. The biotech would need to prove efficacy in something like the 6-minute walk test to determine if their second drug, golodirsen, was actually helping the boys who suffered from the disease.

Testing an unproven theory against the reality of the declining strength these boys typically face in confronting a rare, lethal disease, Woodcock told her colleagues, a new application with hard data in it would give her a chance to determine if eteplirsen was providing a safe benefit to DMD patients and families, and if the slight boost in dystrophin is an accurate marker for the benefit.

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Source: Inato.com

The Sanofi-part­nered start­up find­ing clin­i­cal tri­als for the 90%

Sanofi wanted help finding new trial sites, so a couple years ago they started talking with a startup down the street – who sent them to China.

The Paris-based startup, Inato, was building a platform to expand the pool of patients for clinical trials. Only instead of advertising or matching patients with trials, as other young companies have, Inato was trying to match pharma companies with the vast majority of hospitals that rarely, if ever, host a trial, even if they have patients better suited to a particular study than a marquee name hospital. Call them a trial platform for the 99% – or, by Inato’s calculations, the 90% of sites that are generally ignored in clinical research.

HIV vac­cine re­mains Holy Grail as Sanofi/GSK reg­i­men dis­ap­points in large study

An HIV vaccine regimen comprising experimental components supplied by Sanofi and GlaxoSmithKline has been declared ineffective, underscoring the difficulty of this undertaking and raising the stakes for a mosaic vaccine being tested by a J&J-led coalition.

The National Institute of Allergy and Infectious Diseases has stopped administering vaccines in the HVTN 702 trial after an interim review concluded that it didn’t prevent HIV. The Phase IIb/III trial enrolled 5,407 HIV-negative volunteers in South Africa.

Bio­Mo­tiv, Bris­tol-My­ers al­liance bears fruit with new com­pa­ny: An­teros Phar­ma

As a pharmaceutical accelerator that selects and incubates nascent technology from the halls of academia, BioMotiv is constantly in need of funding. Enter Bristol-Myers Squibb — who along with assimilating the arsenal of Celgene assets it acquired with the $74 billion acquisition of the big biotech company — is looking for fresh meat to bulk up its pipeline.

On Tuesday, BioMotiv and Bristol-Myers unveiled the first company borne out of their 2019 pact: Anteros Pharmaceuticals, which is focused on difficult-to-treat fibrotic and other inflammatory diseases, using technology in-licensed from Yale.

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George Yancopoulos, Columbia via Youtube

Re­gen­eron joins the hunt for an­ti­bod­ies to fight a brew­ing pan­dem­ic as re­searchers whip up new tri­als for an­tivi­rals

Count Regeneron as the latest big biopharma company to pitch in on finding a therapy to combat the Wuhan virus.

Health and Human Services said on Tuesday that they’ll be working with Regeneron to use its antibody platform tech — one of the best in the world — to hunt for a therapy to fight the lethal virus. This is an expansion of a joint project they set up a little more than 2 years ago to hunt for antibodies that worked against Ebola and 9 other viruses, including flu viruses.

Too slow to warn: Sanofi in­dict­ed over old an­ti-epilep­tic drug tied to birth de­fects

Sanofi has been formally charged over Depakine — a decades-old therapy for epilepsy that causes birth defects and impedes neurological development when taken during pregnancy.

Evidence that the compound — sodium valproate — was associated with neurodevelopmental risks emerged in the latter half of the 20th century. In 2014, the EMA re-evaluated the drug, and a year later recommendations that practitioners should no longer prescribe the treatment for women of childbearing age or pregnant women were enforced.

Jorge Conde (Andreessen Horowitz via YouTube)

Q&A with Jorge Conde as An­dreessen Horowitz launch­es $750M bio fund III

When the tech VCs at Andreessen Horowitz entered biotech 4.5 years ago with the $200 million bio fund I, the idea was simple and hubristic: “We’re not going to do biotech,” Vijay Pande said at the time, keeping a16z’s longtime stance. Instead, “the bio fund is really about funding software companies in the bio space.”

In the near-half decade since, they haven’t softened their rhetoric. Pande and general partner Jorge Conde’s frequent blog posts often have the tone of”Burning Man” technofuturists. Talking of a “foundational shift in biology,” “bio-revolution,” and “the meaning of life,” and dropping koans like “what is medicine?” has turned them into the well-financed New Age mystics of an AI-driven and bioengineered future.

Julie Yoo

Today, Andreessen Horowitz is launching bio fund III and putting $750 million behind it – more than funds I and II combined. They’ve added new partners, as they did before fund I and II, picking up technologist and entrepreneur Julie Yoo and Vineeta Agarwala, a GV and Broad Institute alumn. It’ll take much of the same tack as the earlier funds, investing early and occasionally up to Series B, and pouring funds not only into therapeutics, but also diagnostics, synthetic biology and startups bringing biological advances into other sectors, such as agriculture.

But Conde tells Endpoints News that the group has learned a thing or two since fund I. Pande had talked about extending Moore’s law to biology through “digital therapeutics” but they were wrong. It wasn’t just about software and artificial intelligence. It was about the long list of ways how biology was done, how drugs were discovered and how the whole healthcare system functions. It was biotechs that worked both with machine learning and wet labs, and founders conversant in both.

Vineeta Agarwala

Since then, they’ve invested in companies like Insitro that integrate AI as a core but not sole part of a drug development chain and Asimov, which is trying to use AI and other tech systems to design a genome from scratch. They even invested in EQRx, Alexander Borisey’s startup trying to use me-too drugs to change pricing.

In October, Conde, Pande and Yoo published their most soaring blog post yet: “Biology is Eating the World: A Manifesto.” They wrote: “We are at the beginning of a new era, where biology has shifted from an empirical science to an engineering discipline.”

Before the fund’s launch, though, Conde told Endpoints we’re “at the end of the beginning” for that era.

He talked about what they’ve learned since bio I, where biology and biotech is headed and how we’ll know when the convergence between engineering and biology he’s been prophesizing has arrived.

You called this the “end of the beginning” for a new era. What does that mean?

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In­smed’s PhII smokes and sput­ters, but re­searchers her­ald enough sur­pris­ing­ly pos­i­tive da­ta to roar on Wall Street

There was plenty not to like in Insmed’s Phase II readout of an anti-inflammatory drug dubbed INS1007.

The dose response didn’t pan out and in fact, the high dose missed on a key endpoint that could be the pivotal goal needed for an approval. But with several analysts expecting the whole thing to be a bust, investors cheered on evidence of positive efficacy in treating non-cystic fibrosis bronchiectasis and ran up the stock price $INSM by 40%.

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