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IMETELSTAT IN RBC TRANSFUSION-DEPENDENT (TD) LOWER RISK MDS RELAPSED/REFRACTORY TO ERYTHROPOIESIS-STIMULATING AGENTS (ESA) (IMERGE): UPDATED EFFICACY AND SAFETY
Author(s): Pierre Fenaux , Azra Raza , Edo Vellenga , Uwe Platzbecker , Valeria Santini , Irina Samarina , Koen Van Eygen , María Díez-Campelo , Mrinal M. Patnaik , Laurie Jill Sherman , Libo Sun , Helen Varsos , Esther Rose , Aleksandra Rizo , David P. Steensma
(Abstract release date: May 17, 2018) EHA Learning Center. Fenaux P. Jun 17, 2018; 214476
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Type: Oral Presentation
Presentation during EHA23: On Sunday, June 17, 2018 from 08:30 - 08:45
Location: Room A4
Patients with lower-risk MDS that is relapsed/refractory to ESA have few treatment options. The first-in-class telomerase inhibitor imetelstat, which targets cells with short telomere lengths and active telomerase, has the potential to provide clinical benefit in this patient population.
Here we report updated safety and efficacy data from IMerge, an ongoing 2-part, global, phase 2/3 study of imetelstat in RBC TD patients with IPSS Low or Intermediate-1 (Int-1) risk MDS.
The IMerge population includes patients with MDS relapsed/refractory to ESA with a transfusion requirement of ≥4 units over 8 weeks prior to entry and ESA-naïve patients with sEPO >500 mU/mL. In Part 1 (open-label, single-arm), imetelstat 7.5 mg/kg was administered IV every 4 weeks (escalation to 9.4 mg/kg permitted after 3 cycles). The primary endpoint is the rate of RBC transfusion-independence (TI) lasting ≥8 weeks; secondary endpoints include safety, ≥24-week TI rate, time to and duration of TI, and hematologic improvement (HI) rate. Updated results for the first 32 patients enrolled in Part 1 (median follow up, 75.3 weeks) are reported here.
Median age was 68.5 years. 59% of patients were IPSS Low and 41% Int-1. 13 patients (43%) had sEPO>500 mU/mL. 34% had a cytogenetic abnormality, including 22% with del(5q). Prior MDS treatments included ESAs (88%), lenalidomide (38%), and decitabine or azacitidine (HMAs) (25%); 41% were both lenalidomide and HMA naïve and non-del(5q). As of Jan 2018, RBC-TI ≥8-week was achieved in 38% of patients. Median time to onset of TI was 8 weeks with a median duration of TI of 23 weeks. 16% of patients achieved ≥24-week TI, and 63% achieved erythroid HI. Of 13 lenalidomide and HMA naïve and non-del(5q) patients, 54% achieved ≥8-week TI. Median time to onset of TI in the subset was 8 weeks with a median duration of TI of 43 weeks. 31% of these patients had ≥ 24-week TI, and 69% achieved erythroid HI. TI response did not differ based on the presence of ringed sideroblasts or baseline sEPO level (41% [7/17] ≤500 mU/L; 38% [5/13] >500 mU/L). Cytopenias, particularly neutropenia and thrombocytopenia, were the most frequently reported adverse events (AEs) overall and in the lenalidomide/HMA naive and non-del(5q) subset. The subset had a lower incidence of gr ≥3 neutropenia relative to the overall population (54% vs 66%) but a higher rate of gr ≥3 thrombocytopenia (62% vs 53%). Gr 4 neutropenia incidences were 41% overall and 38% for the subset. Overall, 28 patients (88%) had reversible LFT elevations by at least one grade. Four patients had gr 3 worsening of AST and/or ALT (1 also had gr 3 worsening of bilirubin), all of which were reversible. Eleven patients received growth factors during the study for treatment of an AE or ongoing medical history (n=10) or as prophylaxis (n=1).
Safety and efficacy reported here support continued investigation of imetelstat using the current dosing regimen of 7.5 mg/kg every 4 weeks in IPSS Low/Int-1 RBC TD MDS patients relapsed/refractory to ESA. AEs (mostly cytopenias) were predictable, manageable, and reversible. TI was observed in 38% and erythroid HI in 63% of patients with imetelstat therapy, with a 16% durable 24-week TI rate. Patients naïve to lenalidomide and HMAs and who lacked del(5q) had a 54% 8-week TI rate and responses were more durable (31% 24-week TI rate). To further validate these findings, additional patients meeting these criteria have been enrolled and are currently being followed.
Session topic: 10. Myelodysplastic syndromes – Clinical
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Ik begrijp dat patiënten langer in leven blijven, maar blijkbaar zijn er toch heel wat nevenwerkingen.
Kan iemand een beetje begrijpbaar duiden hoe erg die nevenwerkingen zijn? Ik vraag dit omdat "langer leven" in zeer moeilijke omstandigheden misschien niet zo zinvol is en misschien toch een reden kan zijn om heel de studie af te blazen.
De primaire criteria van de clinical trials verwijzen trouwens ook meer naar neveneffecten (zoals toename milt volume),
- Forum actieveling
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- Lid geworden op: 03 Nov 2015 23:35
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De frequentste bijwerkingen waren neutropenie (= tekort aan neutrofiele granulocyten, een type witte bloedcellen) en trombopenie (= tekort aan trombocyten = bloedplaatjes). Als je te weinig neutrofiele granulocyten hebt, loop je een hoog risico op infectie. Als je te weinig bloedplaatjes hebt, dreig je te gaan bloeden (gaande van huid- tot hersenbloedingen). Hoe hoger de graad van de bijwerking, des te ernstiger is ze. Graad 3- en graad 4-neutropenie en -trombopenie waren blijkbaar zeer frequent en dat zijn dus ernstige, potentieel levensbedreigende bijwerkingen. Andere bijwerkingen waren leverteststoornissen: blijkbaar minder ernstig en omkeerbaar na stopzetting van de behandeling.
Dus vanaf 17 jullie kan elke dag de beslissing vallen
Geron logoArnhold LLC acquired a new stake in Geron Co. (NASDAQ:GERN) during the first quarter, according to its most recent disclosure with the SEC. The institutional investor acquired 154,700 shares of the biopharmaceutical company’s stock, valued at approximately $657,000. Arnhold LLC owned approximately 0.10% of Geron at the end of the most recent reporting period.
A number of other hedge funds and other institutional investors have also recently modified their holdings of the business. OMERS ADMINISTRATION Corp bought a new stake in shares of Geron during the 1st quarter valued at $442,000. Swiss National Bank lifted its position in shares of Geron by 13.1% during the 1st quarter. Swiss National Bank now owns 284,400 shares of the biopharmaceutical company’s stock valued at $1,209,000 after acquiring an additional 32,900 shares during the period. DekaBank Deutsche Girozentrale bought a new stake in shares of Geron during the 1st quarter valued at $106,000. Wells Fargo & Company MN lifted its position in shares of Geron by 17.0% during the 1st quarter. Wells Fargo & Company MN now owns 124,353 shares of the biopharmaceutical company’s stock valued at $529,000 after acquiring an additional 18,111 shares during the period. Finally, MetLife Investment Advisors LLC bought a new stake in shares of Geron during the 4th quarter valued at $136,000. 34.46% of the stock is currently owned by hedge funds and other institutional investors.
https://stocknewstimes.com/2018/06/03/a ... -gern.html
Deze quote van een zekere "Andrew" gepost op Yahoo vind ik wel interessant en de moeite om hier te posten:
"The question of why J&J hasn't chosen to buy GERN on the cheap is one of the few legitimate questions the shorts post. Obviously no one on the outside knows the answer to that. It is true that only J&J truly knows the status and effectiveness and even the full potential of imetelstat. And it would make sense to make a BO offer before things picked up, if they felt that positive things were on the way a pre-news BO makes sense. Maybe they have major doubts. They have been burned rather recently with the BO of a company they had claimed had $1B+ potential. Because of that, they might be taking this slow and steady and willing to pay a little more and wait for further confirmation. They may opt in and continue to take it slow and wait for FDA approval and prefer to pay more for something approved than risk paying less for nothing if it doesn't get approved. Even at that point, with the percentage they own, they are in the best position to BO GERN. Or maybe the shorts are right and they don't plan on opting in at all and with the current negative sentiment and low share price, they want to delay the negative news of abandoning a drug they have consistently promoted.
I don't think this is the case though. IMO, the reason they are not buying on the cheap is the same reason the shorts are arguing they are walking. J&J are not only in the best position to know the potential, they are the only ones in that position, better than GERN. At the shareholder meeting we were told that GERN is in the dark and has no update yet, only Jansson and J&J know what's going on. If that is the case then J&J could never get away with a low ball offer. They can't offer to buy GERN out on the cheap and then 6 months later announce monster results or FDA approval. They would get sued by every institution and shareholder post sale and lose. They can't withhold or trade on information they have and control and then buy the company "on the cheap" based on that information. Under this unique relationship, they have no choice but to continue under the agreement and then pay GERN what they are worth based on results. They are too far in at this point to attempt a pre-emptive strike without paying full value. Therefore, they are better off letting this play out and paying fair value based on results. If the FDA approves imetelstat in it's two initial applications its a win for everyone, GERN likely gets a full value BO and JJ gets a FDA approved drug with huge potential. If the FDA does not approve then JJ dodged a bullet and GERN goes to 0. Therefore, the continuation decision is the key to the SP. Yes, is a vote of confidence and a indication of JJ opinion on FDA approval and a non-continuation is a disaster. We should find out in the next few months"