Phar­ma’s sum­mer of punk: Who's play­ing the hits — or tak­ing a hit — on the world in­no­va­tion tour

Before I owned a record label, I used to write a syndicated album review column. One of the series we ran was on the ‘golden streaks’ – the 3 album runs that have tended to define the peak of the careers of most of the ‘classic artists’, from Stevie Wonder to Sinatra to Led Zeppelin. It is possible that we have just witnessed our own industry’s punk period – older artists still selling well, bringing in the crowds, but the power now being in the hands of smaller, self-made artists, who may never feel compelled to ‘sell out’.

The Pharmaceutical Innovation Index measures, scores and celebrates a company’s ability to deliver innovation to patients, by objectively evaluating performance based on a rolling five year period (2013-2018), and operates on the simple premise: if you gave the same molecule to two different companies in early phase, which would make the best of it? This year provides perhaps the strongest indication yet of how unevenly distributed that competency (launching meaningful medicines) is…

Gilead has risen consistently, by one place per year since 2016, to the top of the table, with an outstanding antiviral performance – in 2018, Gilead derived 60% of its revenues from new drugs, more than twice any other company, and 6 times more than the average across the top 30. It should also be noted that Gilead are the only company consistently delivering new drugs that hit what we’d traditionally call ‘blockbuster’ status within 2-3 years of launch.

There is no gaming the Pharmaceutical Innovation Index – success is achieved by discovering and developing meaningfully great medicines and getting those medicines to market, and to patients. What Gilead has achieved is remarkable – they have redefined success for this industry – launching truly meaningful medicines that swing for the bleachers rather than bunt, embracing novelty and originality over incrementalism. However, the Index also shows that successful innovation is not evenly distributed – more than half of the top 30 companies did not achieve an approval in 2018. 2019 also marks the first time in the nine years of PII that J&J have not been in the Top 10, the inverse of Gilead’s journey over that same time. (To be balanced, Gilead haven’t yet proven that they can take antiviral skills into another therapeutic area.)

In a year that showed a record number of new drug approvals at the FDA, it is notable that over half were for rare diseases, and 39 of the 59 FDA approvals went to small- to mid-sized biotech (companies outside of the top 30), suggesting that there are significant innovation and productivity challenges for large pharma in the coming years. It also suggests that, for those 39 companies and their 39 approvals, the most important next step is to prove they can successfully commercialise. As Scott Gottlieb has hinted, it is not the regulators who are holding up the industry’s innovation – maybe it’s the major labels who haven’t yet learned to cope with the indies.

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Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Franz-Werner Haas, CureVac CEO

3 weeks, 3 big in­vest­ments capped by a $213M IPO bring Cure­Vac’s in­stant-cash raise to a cool bil­lion dol­lars

CureVac jumped onto Nasdaq Thursday night, landing with an extra $213 million after selling a batch of shares at $16 a pop. Add in an extra $118 million share purchase by founder Dietmar Hopp — who owns a controlling interest — and another $640 million from deals and the German biotech has raised a cool billion dollars in the space of just 3 weeks.

The company’s stock $CVAC will now start trading this morning, with analysts eager to find out whether the go-go atmosphere on Wall Street will swell the biotech’s share price.

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Martin Shkreli (Shutterstock)

Mar­tin Shkre­li con­tin­ued to or­ches­trate an­ti-com­pet­i­tive schemes for Dara­prim be­hind bars — FTC

Martin Shkreli didn’t just blog, read up on drug development news and run his biotech business with a contraband cell phone in prison. According to the FTC, he was also coordinating the anticompetitive scheme to shield Daraprim — the drug at the center of a price-gouging controversy that earned him the “Pharma Bro” nickname — from generic rivals.

Back in January the FTC, together with New York’s attorney general, launched a federal lawsuit against Shkreli, who’s now serving a 7-year sentence for defrauding investors in his hedge fund, alleging that he effectively created a drug monopoly. While Shkreli’s notorious move to raise the per tablet price of Daraprim from $17.50 to $750 was perfectly legal, the tactics he allegedly deployed to box out competitors weren’t.

NIH director Francis Collins at a Senate Appropriations subcommittee hearing for Operation Warp Speed (Graeme Jennings/Pool via AP Images)

Covid-19 roundup: 'No­vem­ber or De­cem­ber' Collins' best bet on a vac­cine OK; First plas­ma tri­al sug­gests mor­tal­i­ty re­duc­tion

Count NIH director Francis Collins out for any wager that the FDA would authorize a Covid-19 vaccine in October.

The discussion came up during a call with reporters because some states and local governments have been told by the CDC to have vaccination plans ready to go by Oct. 1. Pharma execs, most notably from Pfizer and BioNTech, have raised hopes about a licensure during that month; President Donald Trump last week sounded an optimistic note about having a vaccine on the market “right around” Election Day on Nov. 3 — or possibly before.

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Charlie Silver (Mission Bio)

'We want to be every­where.' Mis­sion Bio rais­es $70M be­hind re­sis­tance-hunt­ing se­quenc­ing plat­form

Charlie Silver wants to look really, really closely at a lot of your cells. And he just got a lot of money to do so.

Silver’s startup, Mission Bio, raised $70 million in a Series C round Thursday led by Novo Holdings. The money, which brings Mission Bio to $120 million raised since its 2012 founding, will be used to advance the single-cell sequencing platform they built to detect early response or resistance to new cancer therapies.

Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.