ImmunityBio's (IBRX) bladder cancer therapy Anktiva, Verona Pharma's (VRNA) non-steroidal
COPD therapy, ensifentrine, and Geron's (GERN) blood cancer therapy, Imetelstat, round out the
list.
Evaluate's top new drug list
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Re: Evaluate's top new drug list
Here's a link.....
https://www.fiercepharma.com/special-re ... nches-2024
10. Imetelstat
By Nick Paul Taylor
Drug: Imetelstat
Company: Geron
Used for: Lower-risk myelodysplastic syndromes
Est. 2028 sales: $737 million
"After more than 30 years of work, Geron is less than six months from a potential landmark approval. The drug candidate, imetelstat, is on the cusp of becoming the first telomerase inhibitor to be authorized in the U.S.
Geron set up shop in 1990 and went public in 1996. Back then, the biotech’s work informed headlines such as “Could immortality be within grasp? Scientists say yes” but hype about the anti-aging potential of telomerase inhibitors was later swamped by costly setbacks. At the end of 2022, Geron’s accumulated deficit stood at $1.4 billion.
The money has supported a 20-year odyssey. Geron began a phase 1 trial of imetelstat, then known as GRN163L, in 2005. A laundry list of clinical trials failed to find a place for the molecule. Geron stopped a phase 2 breast cancer trial in 2012, halted work on solid tumors with short telomeres in 2013, was hit by a clinical hold in 2014, failed an interim analysis in 2016 and lost its Johnson & Johnson deal in 2018.
As the setbacks mounted up, Geron continued to look for a setting where imetelstat could make a mark. The 2017 decision to expand a phase 2 trial to include patients with lower-risk myelodysplastic syndrome (MDS) ultimately provided the breakthrough. Geron reported pivotal data in the setting early last year.
The trial hit its primary endpoint—transfusion independence at eight weeks—and went on to show that some people can go more than one year without needing to receive red blood cells. Geron used the data to file for FDA approval, securing a decision date in June, and set about positioning its molecule to carve out a niche in a market targeted by companies including Bristol Myers Squibb.
Geron CEO John Scarlett explained the pitch on a quarterly results call with investors in November. The biotech has identified several subgroups of patients in which it believes imetelstat has an advantage over the competition. Geron’s estimate of the total addressable U.S. market—$3.5 billion by 2033—suggests there is room for multiple players, and the biotech sees a persistent unmet need.
“This is a market that's seen low competitive intensity and relatively little innovation in the last decade,” Scarlett said. “The National Comprehensive Cancer Network ... published a revised version of its MDS guidelines, which still point to limited treatment options available to hematologists as they manage their transfusion-dependent lower-risk MDS patients.”
Geron wants to win an initial approval in lower-risk MDS and expand into intermediate-2 or high-risk myelofibrosis. A phase 3 trial in myelofibrosis patients who are relapsed or refractory to JAK inhibition is set to reach an interim analysis in the first half of 2025, with the final analysis to follow the next year. The trial could unlock another market that Geron estimates will be worth $3.5 billion in 2031."
https://www.fiercepharma.com/special-re ... nches-2024
10. Imetelstat
By Nick Paul Taylor
Drug: Imetelstat
Company: Geron
Used for: Lower-risk myelodysplastic syndromes
Est. 2028 sales: $737 million
"After more than 30 years of work, Geron is less than six months from a potential landmark approval. The drug candidate, imetelstat, is on the cusp of becoming the first telomerase inhibitor to be authorized in the U.S.
Geron set up shop in 1990 and went public in 1996. Back then, the biotech’s work informed headlines such as “Could immortality be within grasp? Scientists say yes” but hype about the anti-aging potential of telomerase inhibitors was later swamped by costly setbacks. At the end of 2022, Geron’s accumulated deficit stood at $1.4 billion.
The money has supported a 20-year odyssey. Geron began a phase 1 trial of imetelstat, then known as GRN163L, in 2005. A laundry list of clinical trials failed to find a place for the molecule. Geron stopped a phase 2 breast cancer trial in 2012, halted work on solid tumors with short telomeres in 2013, was hit by a clinical hold in 2014, failed an interim analysis in 2016 and lost its Johnson & Johnson deal in 2018.
As the setbacks mounted up, Geron continued to look for a setting where imetelstat could make a mark. The 2017 decision to expand a phase 2 trial to include patients with lower-risk myelodysplastic syndrome (MDS) ultimately provided the breakthrough. Geron reported pivotal data in the setting early last year.
The trial hit its primary endpoint—transfusion independence at eight weeks—and went on to show that some people can go more than one year without needing to receive red blood cells. Geron used the data to file for FDA approval, securing a decision date in June, and set about positioning its molecule to carve out a niche in a market targeted by companies including Bristol Myers Squibb.
Geron CEO John Scarlett explained the pitch on a quarterly results call with investors in November. The biotech has identified several subgroups of patients in which it believes imetelstat has an advantage over the competition. Geron’s estimate of the total addressable U.S. market—$3.5 billion by 2033—suggests there is room for multiple players, and the biotech sees a persistent unmet need.
“This is a market that's seen low competitive intensity and relatively little innovation in the last decade,” Scarlett said. “The National Comprehensive Cancer Network ... published a revised version of its MDS guidelines, which still point to limited treatment options available to hematologists as they manage their transfusion-dependent lower-risk MDS patients.”
Geron wants to win an initial approval in lower-risk MDS and expand into intermediate-2 or high-risk myelofibrosis. A phase 3 trial in myelofibrosis patients who are relapsed or refractory to JAK inhibition is set to reach an interim analysis in the first half of 2025, with the final analysis to follow the next year. The trial could unlock another market that Geron estimates will be worth $3.5 billion in 2031."