Post by icemandios on Nov 15, 2019 18:03:12 GMT
November 15, 2019 07:59 AM ESTUpdated 10:59 AM
John Carroll
R&D
Novartis spinout’s first anti-aging PhIII is a flop, so now they’ll turn to Parkinson’s challenge as shares wilt
Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.
Investigators for the company determined that their Phase III study demonstrated that their TORC1 inhibitor —RTB101 — actually did slightly worse than a placebo in preventing clinically symptomatic respiratory illness among a group of patients 65 and older.
The p value in the study came out at a disastrous 0.65, forcing the biotech to scrap the advanced program and turn to an early-stage study of the same drug combined with sirolimus in Parkinson’s disease —one of the toughest targets in biotech.
The drug, their only disclosed therapy, is also being studied for heart disease and urinary tract disease.
The news decimated the biotech’s shares, sending the stock down 70% in the latest example of the market’s merciless response to biotech failures.
Geoffrey Porges’ note on the trial fiasco had an air of finality to it:
Beyond today’s trading, investors are unlikely to give the company much credit for the company’s further development efforts in this or any other indication, and the timelines for value realization in such indications (eg Parkinson’s disease) are likely to be long (and the cash requirements substantial).
Inhibiting the mTORC1 pathway has been long studied for its ability — in animals — to revitalize an aging immune system and restore biologic function, in part by cleaning up the toxic loads that build up in our cells as we grow older. PureTech thought this would be a great way to launch a company built on anti-aging R&D when they got the drug from NIBR. But the research strategy has proven to be an extremely complex affair.
In resTORbio’s case, the biotech has alternately delighted and disappointed investors. A combination Phase IIb study as a monotherapy for respiratory disease came up with statistically significant data. But that followed a decisive failure for a combination approach they had tried.
Chen Schor
Author
John Carroll
John Carroll
R&D
Novartis spinout’s first anti-aging PhIII is a flop, so now they’ll turn to Parkinson’s challenge as shares wilt
Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.
Investigators for the company determined that their Phase III study demonstrated that their TORC1 inhibitor —RTB101 — actually did slightly worse than a placebo in preventing clinically symptomatic respiratory illness among a group of patients 65 and older.
The p value in the study came out at a disastrous 0.65, forcing the biotech to scrap the advanced program and turn to an early-stage study of the same drug combined with sirolimus in Parkinson’s disease —one of the toughest targets in biotech.
The drug, their only disclosed therapy, is also being studied for heart disease and urinary tract disease.
The news decimated the biotech’s shares, sending the stock down 70% in the latest example of the market’s merciless response to biotech failures.
Geoffrey Porges’ note on the trial fiasco had an air of finality to it:
Beyond today’s trading, investors are unlikely to give the company much credit for the company’s further development efforts in this or any other indication, and the timelines for value realization in such indications (eg Parkinson’s disease) are likely to be long (and the cash requirements substantial).
Inhibiting the mTORC1 pathway has been long studied for its ability — in animals — to revitalize an aging immune system and restore biologic function, in part by cleaning up the toxic loads that build up in our cells as we grow older. PureTech thought this would be a great way to launch a company built on anti-aging R&D when they got the drug from NIBR. But the research strategy has proven to be an extremely complex affair.
In resTORbio’s case, the biotech has alternately delighted and disappointed investors. A combination Phase IIb study as a monotherapy for respiratory disease came up with statistically significant data. But that followed a decisive failure for a combination approach they had tried.
Chen Schor
Chen Schor, co-founder, president and CEO of resTORbio, insisted that the science is solid, adding: “Multiple pre-clinical models have demonstrated that inhibition of TORC1 decreases protein and lipid synthesis, increases lysosomal biogenesis and stimulates the clearance of misfolded protein aggregates, such as toxic synucleins, that cause neuronal toxicity in Parkinson’s disease. We remain committed to exploring the potential benefits of TORC1 inhibition in patients, and we look forward to the data from our Parkinson’s disease trial, which we expect in mid-2020.”
John Carroll
