TEGSEDI provides powerful knockdown of TTR protein, delivering significant and sustained benefits in neuropathy and quality of life
TEGSEDI is an effective treatment choice that allows self-administration at a time and place that works for the patient
BOSTON, MA and CARLSBAD, CA, USA I October 05, 2018 I Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today that the U.S. Food and Drug Administration (FDA) has approved TEGSEDITM (inotersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. TEGSEDI is now approved in the U.S., European Union and Canada.
“TEGSEDI is the first and only RNA-targeting therapeutic that powerfully reduces the production of TTR protein through a once-weekly subcutaneous injection offering patients an effective treatment for people living with polyneuropathy caused by hATTR amyloidosis. We believe this profile will make TEGSEDI an excellent choice for many patients and that it’s self-administration gives the flexibility to treat at a time that works for them which could change the way this progressive and debilitating disease is treated and managed,” said Paula Soteropoulos, chief executive officer at Akcea Therapeutics. “As we execute on our multi-country launch, we are committed to delivering a comprehensive treatment experience for people taking TEGSEDI. Beyond the drug itself, this includes ensuring patients who need this therapy have access to it, offering personalized support programs and enabling patients to administer treatment on their own terms.”
In hATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body. TEGSEDI targets the disease at its source by reducing the production of TTR protein. In the NEURO-TTR study, treatment with TEGSEDI produced up to a 79% mean decrease from baseline in serum TTR protein in patients regardless of TTR mutation, sex, age, or race.
“Although the true incidence is not known of hereditary ATTR amyloidosis with polyneuropathy, there is no doubt that it is currently significantly underdiagnosed,” said Isabelle Lousada, founder and chief executive officer of the Amyloidosis Research Consortium. “This approval represents a significant advancement for the patients, families, caregivers and healthcare professionals in the U.S. who need more options for the medical management of this disease. I am now more optimistic than ever that we can increase awareness of this rare disease, support faster diagnosis and provide better treatment.”
The FDA’s approval of TEGSEDI was based on results from the Phase 3 NEURO-TTR study in patients with hATTR amyloidosis with symptoms of polyneuropathy. Results from that study demonstrated that patients treated with TEGSEDI experienced significant benefit compared to patients treated with placebo across both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression.
TEGSEDI is associated with risk of thrombocytopenia and glomerulonephritis. Enhanced monitoring is required to support early detection and management of these identified risks. For TEGSEDI’s full prescribing information, including boxed warning, please visit www.TEGSEDI.com and please see Important Safety Information below. TEGSEDI is being marketed with a Risk Evaluation and Mitigation Strategy (REMS).
“hATTR amyloidosis is a fatal disease that affects multiple organs and body systems and robs people of so much – the ability to work, the ability to carry out daily tasks – all the things that make them feel in control of their lives,” said Morie Gertz, M.D., hematologist and Chair Emeritus of Internal Medicine at Mayo Clinic. “TEGSEDI has demonstrated a nearly 80% reduction in TTR, which is now a validated approach to treating this disease. The rapid and sustained improvements compared to placebo and reversal in measures of disease seen in a substantial proportion of patients coupled with the independence offered through self injection provide a sense of hope not only to patients, but to their caregivers and families as well.”
To assist hATTR amyloidosis patients in gaining access to TEGSEDI, Akcea has created Akcea ConnectTM, a patient support program made up of dedicated, regionally-based nurse case managers who have a wide range of medical knowledge and experience.This program offers free, private and personalized support to patients and their caregivers and families across the United States. Akcea Connect is now accepting patient enrollment and authorization forms.For more information, please see the contact information for Akcea Connect below.
“hATTR amyloidosis is a devastating disease that takes so much from patients. Akcea is ready to help patients take something back,” said Sarah Boyce, president of Akcea Therapeutics. “We understand that managing a rare disease goes beyond providing a new medicine and our highly skilled team is fully prepared to provide an effective, safe and comprehensive treatment experience. Our industry-leading patient support program, Akcea Connect, is poised to help patients through every stage of their TEGSEDI treatment journey, and we are well advanced in discussions with leading payers to facilitate access. We are excited and ready to execute on our launch plans and bring TEGSEDI to patients.”
“The U.S. approval of TEGSEDI represents another important addition to the expanding Ionis commercial pipeline, a product of 30 years of innovation. We are thrilled to see our efforts result in an important new medicine for people with polyneuropathy caused by hATTR amyloidosis,” said Brett P. Monia, Ph.D., chief operating officer of Ionis Pharmaceuticals. “We are confident that the experienced team at Akcea will deliver on the promise of TEGSEDI. We are grateful to all of the patients and physicians who participated in the TEGSEDI clinical program and who made this landmark approval possible.”
In April, Akcea licensed the worldwide rights to commercialize TEGSEDI from Ionis. Based on the U.S. approval of TEGSEDI, Ionis will receive a $50 million milestone payment that may be made in Akcea common stock or cash. Commercial profits and losses from TEGSEDI will be split 60 percent to Ionis and 40 percent to Akcea.
CONFERENCE CALL
Akcea and Ionis will hold a live webcast today, October 5 at 5:45pm Eastern Time to discuss today’s announcement. Interested parties may listen to the call by dialing (855) 237-2439, passcode 8380957, or access the webcast at www.akceatx.com or www.ionispharma.com. A webcast replay will be available for a limited time at the same address.
ABOUT TEGSEDITM (INOTERSEN)
TEGSEDI was approved by the U.S. Food and Drug Administration (FDA) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. TEGSEDITM (inotersen), discovered and developed by Ionis Pharmaceuticals, is the world’s first and only subcutaneous RNA-targeting drug designed to reduce the production of human transthyretin (TTR) protein. TEGSEDI also received marketing authorization in the European Union and Canada for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).
The approval is based on data from the NEURO-TTR study that was a Phase 3 randomized (2:1), double-blind, placebo-controlled, 15-month, international study in 172 patients with hATTR amyloidosis with symptoms of polyneuropathy. In NEURO-TTR, TEGSEDI demonstrated significant benefit compared to placebo in measures of neuropathy and quality of life as measured by the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score. Patients treated with TEGSEDI experienced similar benefit regardless of subgroups such as age, sex, race, region, Neuropathy Impairment Score (NIS), Val30Met mutation status, and disease stage.
The approval is also based on data from the NEURO-TTR Open Label Extension (OLE) that is an ongoing study for patients who completed the NEURO-TTR study, designed to evaluate the long-term efficacy and safety of TEGSEDI.
ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea is advancing a mature pipeline of six novel drugs, including TEGSEDITM (inotersen), WAYLIVRATM (volanesorsen), AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, all with the potential to treat multiple diseases. All six drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis’ proprietary antisense technology. TEGSEDI is approved in the U.S., E.U. and Canada. WAYLIVRA is under regulatory review for the treatment of familial chylomicronemia syndrome, or FCS, and is currently in Phase 3 clinical development for the treatment of people with familial partial lipodystrophy, or FPL. Akcea is building the infrastructure to commercialize its drugs globally. Akcea is a global company headquartered in Cambridge, Massachusetts. Additional information about Akcea is available at www.akceatx.com.
ABOUT IONIS PHARMACEUTICALS
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, proprietary antisense technology platform with the potential to treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs – such as children and adults with spinal muscular atrophy (SMA). We created SPINRAZA® (nusinersen)* and are proud to have brought new hope to the SMA community by developing the first and only approved treatment for this disease.
Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 drugs with the potential to treat patients with cardiovascular disease, rare diseases, neurological diseases, infectious diseases and cancer. We created TEGSEDI™ (inotersen) the world’s first RNA-targeted therapeutic approved for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin (TTR) amyloidosis (ATTR) that our affiliate Akcea Therapeutics is commercializing. Together with Akcea, we are also bringing new medicines to patients with cardiometabolic lipid disorders.
SOURCE: IONIS Pharmaceuticals
Post Views: 25
TEGSEDI provides powerful knockdown of TTR protein, delivering significant and sustained benefits in neuropathy and quality of life
TEGSEDI is an effective treatment choice that allows self-administration at a time and place that works for the patient
BOSTON, MA and CARLSBAD, CA, USA I October 05, 2018 I Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today that the U.S. Food and Drug Administration (FDA) has approved TEGSEDITM (inotersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. TEGSEDI is now approved in the U.S., European Union and Canada.
“TEGSEDI is the first and only RNA-targeting therapeutic that powerfully reduces the production of TTR protein through a once-weekly subcutaneous injection offering patients an effective treatment for people living with polyneuropathy caused by hATTR amyloidosis. We believe this profile will make TEGSEDI an excellent choice for many patients and that it’s self-administration gives the flexibility to treat at a time that works for them which could change the way this progressive and debilitating disease is treated and managed,” said Paula Soteropoulos, chief executive officer at Akcea Therapeutics. “As we execute on our multi-country launch, we are committed to delivering a comprehensive treatment experience for people taking TEGSEDI. Beyond the drug itself, this includes ensuring patients who need this therapy have access to it, offering personalized support programs and enabling patients to administer treatment on their own terms.”
In hATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body. TEGSEDI targets the disease at its source by reducing the production of TTR protein. In the NEURO-TTR study, treatment with TEGSEDI produced up to a 79% mean decrease from baseline in serum TTR protein in patients regardless of TTR mutation, sex, age, or race.
“Although the true incidence is not known of hereditary ATTR amyloidosis with polyneuropathy, there is no doubt that it is currently significantly underdiagnosed,” said Isabelle Lousada, founder and chief executive officer of the Amyloidosis Research Consortium. “This approval represents a significant advancement for the patients, families, caregivers and healthcare professionals in the U.S. who need more options for the medical management of this disease. I am now more optimistic than ever that we can increase awareness of this rare disease, support faster diagnosis and provide better treatment.”
The FDA’s approval of TEGSEDI was based on results from the Phase 3 NEURO-TTR study in patients with hATTR amyloidosis with symptoms of polyneuropathy. Results from that study demonstrated that patients treated with TEGSEDI experienced significant benefit compared to patients treated with placebo across both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression.
TEGSEDI is associated with risk of thrombocytopenia and glomerulonephritis. Enhanced monitoring is required to support early detection and management of these identified risks. For TEGSEDI’s full prescribing information, including boxed warning, please visit www.TEGSEDI.com and please see Important Safety Information below. TEGSEDI is being marketed with a Risk Evaluation and Mitigation Strategy (REMS).
“hATTR amyloidosis is a fatal disease that affects multiple organs and body systems and robs people of so much – the ability to work, the ability to carry out daily tasks – all the things that make them feel in control of their lives,” said Morie Gertz, M.D., hematologist and Chair Emeritus of Internal Medicine at Mayo Clinic. “TEGSEDI has demonstrated a nearly 80% reduction in TTR, which is now a validated approach to treating this disease. The rapid and sustained improvements compared to placebo and reversal in measures of disease seen in a substantial proportion of patients coupled with the independence offered through self injection provide a sense of hope not only to patients, but to their caregivers and families as well.”
To assist hATTR amyloidosis patients in gaining access to TEGSEDI, Akcea has created Akcea ConnectTM, a patient support program made up of dedicated, regionally-based nurse case managers who have a wide range of medical knowledge and experience.This program offers free, private and personalized support to patients and their caregivers and families across the United States. Akcea Connect is now accepting patient enrollment and authorization forms.For more information, please see the contact information for Akcea Connect below.
“hATTR amyloidosis is a devastating disease that takes so much from patients. Akcea is ready to help patients take something back,” said Sarah Boyce, president of Akcea Therapeutics. “We understand that managing a rare disease goes beyond providing a new medicine and our highly skilled team is fully prepared to provide an effective, safe and comprehensive treatment experience. Our industry-leading patient support program, Akcea Connect, is poised to help patients through every stage of their TEGSEDI treatment journey, and we are well advanced in discussions with leading payers to facilitate access. We are excited and ready to execute on our launch plans and bring TEGSEDI to patients.”
“The U.S. approval of TEGSEDI represents another important addition to the expanding Ionis commercial pipeline, a product of 30 years of innovation. We are thrilled to see our efforts result in an important new medicine for people with polyneuropathy caused by hATTR amyloidosis,” said Brett P. Monia, Ph.D., chief operating officer of Ionis Pharmaceuticals. “We are confident that the experienced team at Akcea will deliver on the promise of TEGSEDI. We are grateful to all of the patients and physicians who participated in the TEGSEDI clinical program and who made this landmark approval possible.”
In April, Akcea licensed the worldwide rights to commercialize TEGSEDI from Ionis. Based on the U.S. approval of TEGSEDI, Ionis will receive a $50 million milestone payment that may be made in Akcea common stock or cash. Commercial profits and losses from TEGSEDI will be split 60 percent to Ionis and 40 percent to Akcea.
CONFERENCE CALL
Akcea and Ionis will hold a live webcast today, October 5 at 5:45pm Eastern Time to discuss today’s announcement. Interested parties may listen to the call by dialing (855) 237-2439, passcode 8380957, or access the webcast at www.akceatx.com or www.ionispharma.com. A webcast replay will be available for a limited time at the same address.
ABOUT TEGSEDITM (INOTERSEN)
TEGSEDI was approved by the U.S. Food and Drug Administration (FDA) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. TEGSEDITM (inotersen), discovered and developed by Ionis Pharmaceuticals, is the world’s first and only subcutaneous RNA-targeting drug designed to reduce the production of human transthyretin (TTR) protein. TEGSEDI also received marketing authorization in the European Union and Canada for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).
The approval is based on data from the NEURO-TTR study that was a Phase 3 randomized (2:1), double-blind, placebo-controlled, 15-month, international study in 172 patients with hATTR amyloidosis with symptoms of polyneuropathy. In NEURO-TTR, TEGSEDI demonstrated significant benefit compared to placebo in measures of neuropathy and quality of life as measured by the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score. Patients treated with TEGSEDI experienced similar benefit regardless of subgroups such as age, sex, race, region, Neuropathy Impairment Score (NIS), Val30Met mutation status, and disease stage.
The approval is also based on data from the NEURO-TTR Open Label Extension (OLE) that is an ongoing study for patients who completed the NEURO-TTR study, designed to evaluate the long-term efficacy and safety of TEGSEDI.
ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea is advancing a mature pipeline of six novel drugs, including TEGSEDITM (inotersen), WAYLIVRATM (volanesorsen), AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, all with the potential to treat multiple diseases. All six drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis’ proprietary antisense technology. TEGSEDI is approved in the U.S., E.U. and Canada. WAYLIVRA is under regulatory review for the treatment of familial chylomicronemia syndrome, or FCS, and is currently in Phase 3 clinical development for the treatment of people with familial partial lipodystrophy, or FPL. Akcea is building the infrastructure to commercialize its drugs globally. Akcea is a global company headquartered in Cambridge, Massachusetts. Additional information about Akcea is available at www.akceatx.com.
ABOUT IONIS PHARMACEUTICALS
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, proprietary antisense technology platform with the potential to treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs – such as children and adults with spinal muscular atrophy (SMA). We created SPINRAZA® (nusinersen)* and are proud to have brought new hope to the SMA community by developing the first and only approved treatment for this disease.
Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 drugs with the potential to treat patients with cardiovascular disease, rare diseases, neurological diseases, infectious diseases and cancer. We created TEGSEDI™ (inotersen) the world’s first RNA-targeted therapeutic approved for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin (TTR) amyloidosis (ATTR) that our affiliate Akcea Therapeutics is commercializing. Together with Akcea, we are also bringing new medicines to patients with cardiometabolic lipid disorders.
SOURCE: IONIS Pharmaceuticals
Post Views: 25
