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

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

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Cy­to­ki­net­ics’ ALS drug fails PhI­II, leav­ing the biotech with a sin­gle late-stage prospect

Cytokinetics’ candidate for the muscle disease amyotrophic lateral sclerosis, or ALS, failed a Phase III trial, the Bay Area biotech announced Friday morning.

At a second interim analysis of the trial, an independent review committee recommended that Cytokinetics discontinue its COURAGE-ALS trial for reldesemtiv, as it “found no evidence of effect” compared to placebo on the primary or key secondary endpoints.

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CHMP gives thumbs-up for We­govy use in ado­les­cents, along with nine new drug rec­om­men­da­tions

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended nine drugs for approval this week while also giving thumbs up for six expanded indications, including Novo Nordisk’s approved obesity medication Wegovy for younger people. Wegovy is already approved as an obesity treatment in the EU for adults, and the new indication would allow prescriptions for adolescents aged 12 and older.

Austin biotech Mol­e­c­u­lar Tem­plates lays off more than 100 staffers as pipeline nar­rows

Molecular Templates is ridding itself of a Phase I HER2 asset and fine-tuning its pipeline to focus on three programs and a preclinical Bristol Myers Squibb collaboration. With the narrowed scope on its so-called engineered toxin bodies, the Austin, TX biotech is laying off about half of its staff.

That’s a little more than 100 employees, per an SEC filing. Molecular’s layoffs, approved by its board Wednesday, add to the dozens of pullbacks in the industry in the first three months of 2023.

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Green­Light re­ceives buy­out of­fer; Apol­lomics com­pletes SPAC merg­er

RNA biotech GreenLight Biosciences has been handed an offer for potential acquisition.

GreenLight said in a release that it has received a non-binding “indication of interest” from Fall Line Endurance Fund to acquire GreenLight’s capital stock for $0.60 per share in cash. The release said any potential agreement between the two parties would depend on certain conditions.

Through a special committee, the biotech will evaluate the offer but added there’s no certainty a deal will go forward. GreenLight will also not make any more announcements until a deal comes through or “otherwise determines” a statement is necessary.

TScan Therapeutics' departing CEO David Southwell and CSO/COO Gavin MacBeath

TCR up­start an­nounces CEO ex­it, with CSO now act­ing re­place­ment

A public T cell biotech’s chief executive has decided to leave the company.

TScan Therapeutics said Friday morning that CEO David Southwell stepped down earlier this week, leaving both his chief executive and board member roles. Filling in is Gavin MacBeath, the company’s CSO and COO. He became the acting CEO on Tuesday, and will continue to remain CSO and COO, TScan’s announcement read.

Aptinyx eval­u­ates fu­ture of the com­pa­ny fol­low­ing two failed tri­als, 60% lay­offs

This year has been tough for Aptinyx — two failed trials, a 60% cut in its workforce, and now the company has brought on a firm to help evaluate the future of the company.

The press release noted it’s working with the firm Ladenburg Thalmann as its financial advisor to assist in exploring and evaluating “strategic alternatives” — a process that a growing group of struggling biotechs has embarked on, sometimes ending in a merger, asset sale or wind-down.

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Sar­to­rius to ac­quire French man­u­fac­tur­er for $2.6B+ in cell and gene ther­a­py play

The German life science group Sartorius will be picking up French contract manufacturer Polyplus for the price of €2.4 billion, or $2.6 billion.

On Friday, Sartorius announced the acquisition through its French subgroup, Sartorius Stedim Biotech, which will be acquiring Polyplus from private investors ARCHIMED and WP GG Holdings IV. Polyplus has 270 employees and produces materials and components that go into making viral vectors that are used in cell and gene therapies. This includes DNA/RNA reagents as well as plasmid DNA. Polyplus has locations in France, Belgium, China and the US.