Think your odds of R&D suc­cess are mis­er­able? MIT’s An­drew Lo might have a sur­prise for you

Maybe the odds of suc­cess in a clin­i­cal tri­al aren’t as mis­er­able as just about every­one in the bio­phar­ma R&D sec­tor as­sumes.

MIT pro­fes­sor An­drew Lo and his team crunched the re­sults on more than 185,000 clin­i­cal tri­als in­volv­ing 21,000 com­pounds — cour­tesy of some new tech­niques in Big Da­ta analy­sis — and found that near­ly 14% of the drugs that make it to the clin­ic ac­tu­al­ly go on to an ap­proval.

Com­pare that to the 5% suc­cess rate that As­traZeneca just cit­ed in eval­u­at­ing its own suc­cess rate — or the 9.6% fig­ure cit­ed by BIO and Bio­med­track­er that of­ten gets bandied about — and you’ll see Lo is com­ing up here with a sig­nif­i­cant­ly high­er suc­cess ra­tio than the in­dus­try is used to see­ing.

Why is this im­por­tant? Lo is a not­ed ex­pert in the field of biotech risk, and he want­ed to break out the num­bers on which pro­grams had the best rate of suc­cess, and what tracked worse, in or­der to help ex­ecs bet­ter un­der­stand the odds they faced. And the num­ber bears a big in­flu­ence on the whole dis­cus­sion about the in­dus­try’s ROI, which by all ac­counts has been shrink­ing over the past decade.

Lo, the di­rec­tor of MIT’s Lab­o­ra­to­ry for Fi­nan­cial En­gi­neer­ing, puts it this way:

One of the main re­spon­si­bil­i­ties of in­vestors and phar­ma ex­ec­u­tives is risk man­age­ment, hence they need to know what the chances are that a com­pound will tran­si­tion from Phase 1 to Phase 2 to Phase 3 and, ul­ti­mate­ly, re­ceive FDA ap­proval. With­out ac­cu­rate and time­ly es­ti­mates, re­sources may be mis­al­lo­cat­ed and fi­nan­cial re­turns may be mis­judged, which leads to high­er de­vel­op­ment costs, high­er-priced drugs, and lost op­por­tu­ni­ties for in­vestors and, more im­por­tant­ly, pa­tients.

Top marks go to in­fec­tious dis­ease vac­cines, which had a one in three chance of suc­cess. Can­cer drugs had an aw­ful 3.4% suc­cess rate.

That too, is a fig­ure that will like­ly turn a few heads in the R&D game. Can­cer has been at­tract­ing the li­on’s share of the in­vest­ment cash that’s been fun­neled in­to R&D cir­cles in re­cent years, by every mea­sure. And in­deed Lo notes that with the ar­rival of the I/O drugs com­mand­ing so much at­ten­tion, the suc­cess rate climbed in 2015 to 8.3%.

And Lo plans to keep on track­ing the num­bers for the in­dus­try, of­fer­ing some help in set­ting the odds for suc­cess.

“It’s kind of like the dif­fer­ence be­tween dri­ving with GPS to­day ver­sus dri­ving 20 years ago when maps and friends were the on­ly nav­i­ga­tion­al tools at our dis­pos­al,” Lo ob­serves. “Our goal is to show all stake­hold­ers the lay of the land so that they can make more in­formed de­ci­sions about where and how to di­rect their re­sources.”

But not every­one is sat­is­fied that Lo has cap­tured the right ap­proach.


Im­age: An­drew Lo. MIT Sloan School

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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FDA ap­proves the third NMOSD drug in 18 months as Roche/Genen­tech beefs up its port­fo­lio of drugs for neu­ro­log­i­cal dis­or­ders

There were no FDA approved treatments for neuromyelitis optica spectrum disorder at the start of 2019. Now, as of Friday, there are three.

The latest entrant to the market is the Roche/Genentech drug satralizumab after US regulators gave it the thumbs up late Friday. An IL-6 inhibitor, the drug joins Alexion’s Soliris and AstraZeneca spinout Viela Bio’s Uplizna. The annual cost of satralizumab — which will hit the market as Enspryng — will be $190,000 for 13 doses, a Genentech spokesperson said, though the first year of treatment requires 15 doses and cost about $220,000.

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Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for Covid-19 vac­cine -- shares rock­et up

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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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.

Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 14. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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