- ACH-5228 delivers near complete and sustained inhibition of the complement alternative pathway
- Twice-daily oral administration to move forward in Phase 2 trials
- Investigational New Drug (IND) application to be submitted in fourth quarter of 2019
BLUE BELL, PA, USA I July 22, 2019 I Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN), a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients and families affected by complement-mediated diseases, today announced results from their Phase 1 multiple ascending dose (MAD) study with ACH-5228 outside of the United States.
In the randomized Phase 1 MAD study, ACH-5228 was administered to 43 healthy volunteers. Study subjects received oral doses of ACH-5228 ranging from 40 mg to 200 mg twice a day (BID) for fourteen days. The study also included a single dose cohort of 240 mg. The results demonstrated that ACH-5228, when dosed 120 mg BID or higher, achieved near complete and sustained Alternative Pathway (AP) inhibition with a mean value of >95% at steady state concentrations as measured by AP Hemolysis and AP Wieslab assays.
The study demonstrated that ACH-5228 was generally well tolerated over the dose ranges tested, which include the doses expected to be evaluated in Phase 2 trials. The Company expects to submit an IND application to the U.S. Food and Drug Administration supporting clinical development for ACH-5228 in the fourth quarter of 2019.
“We are pleased the ACH-5228 Phase 1 MAD data exceeded our expectations of our second-generation oral small molecule factor D inhibitor. With its improved potency and longer durability of effect, we believe that oral ACH-5228 has the potential to be the best-in-class alternative pathway inhibitor. Our plan is to advance ACH-5228 into Phase 2 clinical trials in multiple diseases. Danicopan, our first-generation alternative pathway inhibitor, validated factor D as a target and we plan to begin a Phase 3 registrational study in early 2020,” said Joe Truitt, Chief Executive Officer of Achillion Pharmaceuticals.
An updated corporate presentation which refers to the Phase 1 MAD results is available in the “Investor & News” section of Achillion’s website: http://ir.achillion.com/events-and-presentations.
About the Achillion Complement Factor D Portfolio
Achillion has leveraged its internal discovery capabilities and a novel complement-related platform to develop oral small molecule drug candidates that are inhibitors of complement factor D. Factor D is an essential serine protease involved in the alternative pathway (AP) of the complement system, a part of the innate immune system. Achillion’s complement platform is focused on seeking to advance oral small molecules that inhibit the AP and can potentially be used in the treatment of immune-related diseases in which complement AP plays a critical role. Potential indications currently being evaluated for these compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN).
About Achillion Pharmaceuticals
Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN) is a clinical-stage biopharmaceutical company focused on advancing its oral small molecule complement inhibitors into late-stage development and commercialization. Research has shown that an overactive complement system plays a critical role in multiple disease conditions including the therapeutic areas of nephrology, hematology, ophthalmology and neurology. Achillion is initially focusing its drug development activities on complement-mediated diseases where there are no approved therapies or where existing therapies are inadequate for patients. Potential indications being evaluated for its compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and immune complex membranoproliferative glomerulonephritis (IC-MPGN). Each of the product candidates in the Company’s oral small molecule portfolio was discovered in its laboratories and is wholly owned. To achieve its goal of advancing its investigational product candidates into Phase 3 clinical trials and commercialization, the Company plans to work closely with key stakeholders including healthcare professionals, patients, regulators and payors.
More information is available at http://www.achillion.com.
SOURCE: Achillion Pharmaceuticals
Post Views: 40
- ACH-5228 delivers near complete and sustained inhibition of the complement alternative pathway
- Twice-daily oral administration to move forward in Phase 2 trials
- Investigational New Drug (IND) application to be submitted in fourth quarter of 2019
BLUE BELL, PA, USA I July 22, 2019 I Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN), a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients and families affected by complement-mediated diseases, today announced results from their Phase 1 multiple ascending dose (MAD) study with ACH-5228 outside of the United States.
In the randomized Phase 1 MAD study, ACH-5228 was administered to 43 healthy volunteers. Study subjects received oral doses of ACH-5228 ranging from 40 mg to 200 mg twice a day (BID) for fourteen days. The study also included a single dose cohort of 240 mg. The results demonstrated that ACH-5228, when dosed 120 mg BID or higher, achieved near complete and sustained Alternative Pathway (AP) inhibition with a mean value of >95% at steady state concentrations as measured by AP Hemolysis and AP Wieslab assays.
The study demonstrated that ACH-5228 was generally well tolerated over the dose ranges tested, which include the doses expected to be evaluated in Phase 2 trials. The Company expects to submit an IND application to the U.S. Food and Drug Administration supporting clinical development for ACH-5228 in the fourth quarter of 2019.
“We are pleased the ACH-5228 Phase 1 MAD data exceeded our expectations of our second-generation oral small molecule factor D inhibitor. With its improved potency and longer durability of effect, we believe that oral ACH-5228 has the potential to be the best-in-class alternative pathway inhibitor. Our plan is to advance ACH-5228 into Phase 2 clinical trials in multiple diseases. Danicopan, our first-generation alternative pathway inhibitor, validated factor D as a target and we plan to begin a Phase 3 registrational study in early 2020,” said Joe Truitt, Chief Executive Officer of Achillion Pharmaceuticals.
An updated corporate presentation which refers to the Phase 1 MAD results is available in the “Investor & News” section of Achillion’s website: http://ir.achillion.com/events-and-presentations.
About the Achillion Complement Factor D Portfolio
Achillion has leveraged its internal discovery capabilities and a novel complement-related platform to develop oral small molecule drug candidates that are inhibitors of complement factor D. Factor D is an essential serine protease involved in the alternative pathway (AP) of the complement system, a part of the innate immune system. Achillion’s complement platform is focused on seeking to advance oral small molecules that inhibit the AP and can potentially be used in the treatment of immune-related diseases in which complement AP plays a critical role. Potential indications currently being evaluated for these compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN).
About Achillion Pharmaceuticals
Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN) is a clinical-stage biopharmaceutical company focused on advancing its oral small molecule complement inhibitors into late-stage development and commercialization. Research has shown that an overactive complement system plays a critical role in multiple disease conditions including the therapeutic areas of nephrology, hematology, ophthalmology and neurology. Achillion is initially focusing its drug development activities on complement-mediated diseases where there are no approved therapies or where existing therapies are inadequate for patients. Potential indications being evaluated for its compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and immune complex membranoproliferative glomerulonephritis (IC-MPGN). Each of the product candidates in the Company’s oral small molecule portfolio was discovered in its laboratories and is wholly owned. To achieve its goal of advancing its investigational product candidates into Phase 3 clinical trials and commercialization, the Company plans to work closely with key stakeholders including healthcare professionals, patients, regulators and payors.
More information is available at http://www.achillion.com.
SOURCE: Achillion Pharmaceuticals
Post Views: 40
