From Global Newswire
Reata Provides Program Update on Phase 2 Rare Renal Clinical Trials
First patient enrolled for all cohorts of PHOENIX
First data from PHOENIX expected 2H18
Retained benefit analysis from Phase 2 portion of CARDINAL expected 3Q18
IRVING, Texas, Feb. 27, 2018 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. (Nasdaq:RETA) (Reata or Company), a clinical-stage biopharmaceutical company, today provided guidance on the timing of data announcements from the ongoing Phase 2 PHOENIX and CARDINAL trials of bardoxolone methyl (“bardoxolone”) in rare forms of chronic kidney disease (“CKD”).
The Phase 2 PHOENIX program is studying bardoxolone in patients with autosomal dominant polycystic kidney disease (“ADPKD”), IgA nephropathy, focal segmental glomerulosclerosis (“FSGS”), and CKD associated with type 1 diabetes. Approximately 25 patients per cohort will receive bardoxolone open-label, orally, once-daily for 12 weeks. The purpose of this study is to determine the safety and efficacy of bardoxolone, and the primary efficacy endpoint is change from baseline in eGFR after 12 weeks of treatment. Each cohort of patients is being independently enrolled and analyzed, and each has now enrolled at least one patient. The Company anticipates that initial data from one or more PHOENIX cohorts will be released during the second half of 2018.
CARDINAL is a Phase 2/3 study of bardoxolone in patients with CKD caused by Alport syndrome. The Phase 2 portion of CARDINAL enrolled a total of 30 patients to assess the safety and efficacy of once-daily, oral administration of bardoxolone, and its primary efficacy endpoint was change from baseline in estimated glomerular filtration rate (eGFR) at week 12. Full primary endpoint results from the study were reported in November 2017 after all patients had reached week 12. Patients remain in the study for up to two years, and eGFR will be measured at 52 weeks following 48 weeks of treatment and 4 weeks of drug withdrawal (“retained benefit”). The Company expects to report the week 52 retained benefit analysis from this study in the third quarter of this year.
Results from the 52-week retained benefit analysis are relevant to the ongoing Phase 3 portion of CARDINAL, a double-blind, placebo-controlled trial enrolling up to 150 patients worldwide. This Phase 3 study can support accelerated approval by the FDA based upon an improvement in eGFR following 48 weeks of once-daily treatment and 4 weeks of drug withdrawal. After this retained benefit analysis, patients will continue on their original study treatment for another 48 weeks, and full approval can be supported by a retained benefit at 104 weeks following a second 4-week drug withdrawal. Prior trials in patients with other forms of CKD have demonstrated that improvements in eGFR are durable for up to two years, and the change in eGFR after 12 weeks correlates with changes at one year on-treatment and post-withdrawal.
“Diverse forms of CKD are driven by a common final set of inflammatory pathways that bardoxolone targets,” said Colin Meyer, M.D., Chief Medical Officer of Reata. “Treatment with bardoxolone has resulted in clinically meaningful increases in kidney function in patients with Alport syndrome, CKD caused by type 2 diabetes, and CKD associated with pulmonary hypertension, and we hope to demonstrate similar efficacy in these additional types of CKD being studied in PHOENIX. We anticipate that bardoxolone may complement commonly used therapies that modestly affect progression in these diseases, which have no FDA-approved treatments.”
About Bardoxolone Methyl
Bardoxolone methyl is an experimental, oral, once-daily activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. In addition to CARDINAL and PHOENIX, bardoxolone methyl is currently being studied in CATALYST, a Phase 3 study for the treatment of connective tissue disease associated pulmonary arterial hypertension. The FDA has granted orphan designation to bardoxolone methyl for the treatment of Alport syndrome and pulmonary arterial hypertension.
PKD Fundraising
From Auburn Journal, by Aurora Sain
Corks for a cure
Four families volunteering for the Sacramento Chapter of the PKD Foundation, who are emotionally and personally touched by polycystic kidney disease, are hosting the third annual Corks for a Cure.
The events mission is to raise funds and awareness for polycystic kidney disease (PKD), in which there is no treatment and no cure.
“This is such a fun and meaningful event and I hope you'll help us raise awareness and encourage our community to attend,” said Auburn resident Valen Keefer.
One Placer County family, the Lusby’s, are helping to double the impact of the event this year by matching dollar for dollar raised up to $25,000.
Kari Lusby’s 9-year-old son, Honor, has been enduring chronic pain since the age of 2 and it was not until 2013 that they discovered it was polycystic kidney disease. After Kari and her family learned that there is no treatment or cure for PKD, they established Crusade for Cures Foundation. The Lusbys are determined to end PKD so that Honor and millions of others battling this disease can live happy and healthy lives. They are excited to support this year’s Corks for a Cure event and help double the impact with all donations going to fund life-saving medical research.
Polycystic kidney disease is one of the most common, life-threatening genetic diseases. PKD is when fluid-filled cysts develop and enlarge in both kidneys, eventually leading to kidney failure. The average size of a normal kidney is a human fist, polycystic kidneys can get much larger, some getting as large as a football and weighing up to 30 pounds each.
More than 50 percent of people with PKD will develop kidney failure by the age of 50. Once a person has kidney failure, dialysis and transplantation are the only options to treat the damage the disease has caused. Parents have a 50 percent chance of passing the disease to each of their children.
PKD can also develop spontaneously; about 10 percent of the people diagnosed have no family history of the disease. The Sacramento Chapter Coordinator of the PKD Foundation and Corks for a Cure organizer, Julia Adams of Fair Oaks, knows this firsthand. Her daughter, Elizabeth, developed PKD spontaneously and was diagnosed when she was 2 years old and faced kidney failure in her early 20s. Elizabeth’s dad, Rick, was her living kidney donor.
“I started volunteering for the PKD Foundation and raising funds so other families don't have to hear, "Your child has PKD, there is no treatment and no cure,” Adams said.
The event will feature a silent auction, wine, hors d’ oeuvres and dancing.
Living with PKD
The events mission is to raise funds and awareness for polycystic kidney disease (PKD), in which there is no treatment and no cure.
“This is such a fun and meaningful event and I hope you'll help us raise awareness and encourage our community to attend,” said Auburn resident Valen Keefer.
One Placer County family, the Lusby’s, are helping to double the impact of the event this year by matching dollar for dollar raised up to $25,000.
Kari Lusby’s 9-year-old son, Honor, has been enduring chronic pain since the age of 2 and it was not until 2013 that they discovered it was polycystic kidney disease. After Kari and her family learned that there is no treatment or cure for PKD, they established Crusade for Cures Foundation. The Lusbys are determined to end PKD so that Honor and millions of others battling this disease can live happy and healthy lives. They are excited to support this year’s Corks for a Cure event and help double the impact with all donations going to fund life-saving medical research.
Polycystic kidney disease is one of the most common, life-threatening genetic diseases. PKD is when fluid-filled cysts develop and enlarge in both kidneys, eventually leading to kidney failure. The average size of a normal kidney is a human fist, polycystic kidneys can get much larger, some getting as large as a football and weighing up to 30 pounds each.
More than 50 percent of people with PKD will develop kidney failure by the age of 50. Once a person has kidney failure, dialysis and transplantation are the only options to treat the damage the disease has caused. Parents have a 50 percent chance of passing the disease to each of their children.
PKD can also develop spontaneously; about 10 percent of the people diagnosed have no family history of the disease. The Sacramento Chapter Coordinator of the PKD Foundation and Corks for a Cure organizer, Julia Adams of Fair Oaks, knows this firsthand. Her daughter, Elizabeth, developed PKD spontaneously and was diagnosed when she was 2 years old and faced kidney failure in her early 20s. Elizabeth’s dad, Rick, was her living kidney donor.
“I started volunteering for the PKD Foundation and raising funds so other families don't have to hear, "Your child has PKD, there is no treatment and no cure,” Adams said.
The event will feature a silent auction, wine, hors d’ oeuvres and dancing.
Living with PKD
From Goldendale Sentinal, By Max Erikson
Goldendale resident Joanne Davenport has been struggling with Polycystic Kidney Disease (PKD) for many years and is currently one of the 100,000 people nationwide on a waiting list that one day could provide a donated kidney to save her life.
PKD is a hereditary disease that causes numerous fluid-filled cysts to grow in the kidneys. As the cysts grow, it damages the kidney function to the point of kidney failure, also known as renal disease. Kidneys are the organs in the body that filter blood, maintain healthy fluid levels, help make red blood cells, and help keep blood pressure under control.
According to the National Kidney Foundation, 600,000 people in the United States are currently fighting the disease, and one in three American adults are at risk for PKD. Risk factors include having diabetes, high blood pressure, family history of kidney failure, and being age 60 or older.
Davenport has lived in Goldendale for 26 years and was a License Practical Nurse (LPN) at Klickitat Valley Health for 14 years before she got sick. For Davenport, and many others, a kidney transplant is their best hope for living a longer healthier life, but finding a donor with the correct blood type is difficult.
“I have known I’ve had the disease since I was about 30,” Davenport says. “But when I turned 40, it really started to progress. After the last five years of fighting it, I’m at the point where if I don’t find a donor, I will need to start dialysis.”
There are two ways a person with PKD can receive a kidney for transplant. One way is to be on the waiting list to receive a kidney from a deceased organ donor. However, the average wait time for a kidney from a deceased person is three to five years. Many with PKD don’t live long enough to exercise that option.
The other option is to receive a kidney from a person who is alive and willing to donate. That is known as a living donation. Living organ donation programs were developed as a direct result of the critical shortage of deceased donors. Living donations give the best chance at survival for individuals waiting for a transplant, and oftentimes it can be a donation from a close friend or family member. In 2015 6000 living donations were made in the United States.
Living donations are a faster alternative if the right match can be found, which requires matching the correct blood type with the donor and the recipient. Davenport has recently made a public request to the Goldendale community to see if there is anybody interested in becoming a living donor or willing to help find someone who will.
“I’m hoping that there will be someone with a gracious heart willing to help and give me a chance to live,” Davenport says.
Davenport has been working with the Virginia Mason Medical Center in Seattle that has been providing kidney transplants since 1972. Virginia Mason has a living donation program, and for those who might be interested in learning more about donating, Davenport encourages people to visit their website at http://www.virginiamason.org/living-donation.
From Lynn Journal, Boston
John Nucci Faces the Biggest Battle of His Life:Respected Former Suffolk County Clerk Magistrate in Need of a Kidney Transplant
PKD is a hereditary disease that causes numerous fluid-filled cysts to grow in the kidneys. As the cysts grow, it damages the kidney function to the point of kidney failure, also known as renal disease. Kidneys are the organs in the body that filter blood, maintain healthy fluid levels, help make red blood cells, and help keep blood pressure under control.
According to the National Kidney Foundation, 600,000 people in the United States are currently fighting the disease, and one in three American adults are at risk for PKD. Risk factors include having diabetes, high blood pressure, family history of kidney failure, and being age 60 or older.
Davenport has lived in Goldendale for 26 years and was a License Practical Nurse (LPN) at Klickitat Valley Health for 14 years before she got sick. For Davenport, and many others, a kidney transplant is their best hope for living a longer healthier life, but finding a donor with the correct blood type is difficult.
“I have known I’ve had the disease since I was about 30,” Davenport says. “But when I turned 40, it really started to progress. After the last five years of fighting it, I’m at the point where if I don’t find a donor, I will need to start dialysis.”
There are two ways a person with PKD can receive a kidney for transplant. One way is to be on the waiting list to receive a kidney from a deceased organ donor. However, the average wait time for a kidney from a deceased person is three to five years. Many with PKD don’t live long enough to exercise that option.
The other option is to receive a kidney from a person who is alive and willing to donate. That is known as a living donation. Living organ donation programs were developed as a direct result of the critical shortage of deceased donors. Living donations give the best chance at survival for individuals waiting for a transplant, and oftentimes it can be a donation from a close friend or family member. In 2015 6000 living donations were made in the United States.
Living donations are a faster alternative if the right match can be found, which requires matching the correct blood type with the donor and the recipient. Davenport has recently made a public request to the Goldendale community to see if there is anybody interested in becoming a living donor or willing to help find someone who will.
“I’m hoping that there will be someone with a gracious heart willing to help and give me a chance to live,” Davenport says.
Davenport has been working with the Virginia Mason Medical Center in Seattle that has been providing kidney transplants since 1972. Virginia Mason has a living donation program, and for those who might be interested in learning more about donating, Davenport encourages people to visit their website at http://www.virginiamason.org/living-donation.
From Lynn Journal, Boston
John Nucci Faces the Biggest Battle of His Life:Respected Former Suffolk County Clerk Magistrate in Need of a Kidney Transplant
Throughout his political career, whether it was running for Boston School Committee, City Council or Suffolk County clerk magistrate, John Nucci has relied on the help of others to help him win.
Now, Nucci needs the help of others more than ever to help him win the biggest battle of his life.
In his 30s, after the passing of his father following complications of Polycystic Kidney Disease, Nucci found out that he had inherited the same genetic disorder where the renal tubules become structurally abnormal, resulting in the development and growth of multiple cysts within the kidney.
The diagnosis was grim and for the last three decades Nucci lived knowing that someday his kidneys would begin shutting down.
“I was tested in my 30s to find out if I had inherited the disease and I was told then that I did in fact have cysts on my kidneys,” said Nucci. “As these cysts form over the years your kidney function gets lower and lower. My doctors at MassGeneral have been watching it every year since I was in my 30s and in the past year my levels have been dropping fast. That’s the nature of this disease. Once you’re diagnosed you’re never at full kidney function but you can live at 30 percent kidney function for years. Once it drops down to about 15 percent is when they say ‘it’s time.'”
For Nucci ‘it’s time’ meant starting the process of finding a living kidney donor in order to potentially avoid dialysis treatments and the further deterioration of his failing kidneys.
“I’m months away from dialysis,” said Nucci. “It’s inevitable, it’s going to come. But while dialysis is a life saver it’s not a good quality of life. The other problem is that if I have to go that route the longer I’m on dialysis waiting for a donor the less likely a kidney transplant will work or last.”
No stranger to rallying the troops in Eastie and citywide, Nucci, his wife, Peggy, and their three sons, John, Michael and Danny have launched a campaign in local Boston newspapers, on social media and through the relationships Nucci has cultivated over the years to find a living donor match.
Nucci’s wife and his boys have been quarterbacking the campaign to find a living donor as soon as possible. His three boys all tested positive for Polycystic Kidney Disease and were automatically eliminated as donors so they switched gears and began reaching out to other friends and family to find a match.
“I’m not social media savvy so my wife and the boys have been doing a tremendous job getting the word out on Facebook, making phone calls and reaching out to anyone and everyone,” said Nucci. “Finding a living donor is not the hard part and I have had dozens of people step up and get tested and for that I’m beyond grateful. A lot of people have already gone through the process of extensive testing at Mass General but for one reason or another they were eliminated. The problem isn’t so much finding someone who wants to donate it is finding a match. A direct donor has to be a direct blood type (Nucci is Type O) and Mass General is very diligent making sure that the donor is healthy and their kidneys are healthy.”
Nucci said waiting for a deceased donor could take seven to nine years and at his age the direct donor is really the only viable option.
“It’s a race against time,” he said. “I’m ready in terms of my own health. I am a healthy candidate for the transplant it’s just finding the match.”
In the meantime, his son, John, is running the Hyannis Sprint Triathlon to raise money and awareness for Polycystic Kidney Disease.
“In addition to raising money for this great cause, I’m also hoping to get the word out to as many potential donors as possible,” said John. “I know that it’s a huge ask, and it’s not something I would put out there if it wasn’t absolutely necessary, but we’re hoping that someone out there is willing and able to help him. I didn’t even know what a reasonable amount to target would be, and I’m honestly overwhelmed by the support. We’re raising money and getting the word out to so many more people and potential donors than my family and I possibly could’ve on our own, and it seriously means the world to all of us.”
Nucci’s wife Peggy has also been overwhelmed by the amount of support so far.
“I really want everyone to know how much we appreciate everyone’s willingness to help out with our efforts to find the matching kidney donor which John truly needs right now,” said Peggy.
For now, Nucci is trying not to think to much of what lies ahead and focus solely on coming out of this battle victorious, like he did so many times in his political career.
“I’m convinced I’m going to beat this,” said Nucci. “I have never won anything in my life without the help of my friends. My entire political life was spent relying on friends and supporters to win, and this is no different. In many ways it’s like another campaign and I’m going to need that support to win.”
If you or anyone you know is interested in becoming a donor, there is a quick and confidential online screening form that can be found at www.mghlivingdonors.org.
Dialysis Technology
From Medical Design & Outsourcing, By Danielle Kirsh
How self-serve medical devices could make patients feel in control
Intimidating medical devices can cause anxiety for patients — in the same way that people can experience “white coat hypertension” when seeing a doctor.
Devices that are smaller and more patient-centered could help patients feel better about their treatment and make them feel like they’re in charge, according to Leslie Trigg, CEO at Outset Medical (San Jose, Calif.).
“Because you are in control of it, it’s a different experience, even if it takes the same amount of time. It’s really about control. I think when you strip that all away, what really matters to us as human beings is the value we place on being independent and in control of our own destiny. And that’s really on a human level what the driving philosophy is behind Tablo,” Trigg said.
The Tablo is Outset Medical’s 3-foot-high kidney dialysis machine. It makes clean water, produces dialysate, takes blood pressure and delivers medication — all in one compact design. The machine is no taller than a desk and features a touchscreen interface meant to make treatment easier in clinics and hospitals. Its low-profile design is much smaller than other dialysis machines that require special filtration centers and large machines in the office.
“Tablo was launched in some of the existing 6,500 dialysis clinics [in the U.S.] to enable patients to do dialysis on their own. And this idea – we call in-center self-care – is really akin to self-serve dialysis. We kind of just borrowed on the concept of self-serve in retail,” Trigg said. “We are borrowing from that retail concept of self-care and bringing it into the dialysis setting because the system is so easy to use for the average patient. This is an opportunity for the patient to come in and set up Tablo on their own and manage their treatment.”
While Tablo is not currently available for home use, personalizing treatment and making it convenient helps patients feel at ease with their conditions. There are certain chemotherapy treatments that can be done at home, and the National Institutes of Health reports that it can improve patient outcomes and improve the quality of care.
Bringing the treatment closer to the patient is important since people are living longer and it provides a cost-effective solution to different treatments.
“People are living longer,” Trigg said. “If people are able to manage cancer as a chronic disease rather than an acute terminal episode, you have an opportunity – and obviously a responsibility – to make those sorts of chronic [therapies] that are maximumly cost-effective for patients and maximumly cost efficient for payers and providers.”
Now, Nucci needs the help of others more than ever to help him win the biggest battle of his life.
In his 30s, after the passing of his father following complications of Polycystic Kidney Disease, Nucci found out that he had inherited the same genetic disorder where the renal tubules become structurally abnormal, resulting in the development and growth of multiple cysts within the kidney.
The diagnosis was grim and for the last three decades Nucci lived knowing that someday his kidneys would begin shutting down.
“I was tested in my 30s to find out if I had inherited the disease and I was told then that I did in fact have cysts on my kidneys,” said Nucci. “As these cysts form over the years your kidney function gets lower and lower. My doctors at MassGeneral have been watching it every year since I was in my 30s and in the past year my levels have been dropping fast. That’s the nature of this disease. Once you’re diagnosed you’re never at full kidney function but you can live at 30 percent kidney function for years. Once it drops down to about 15 percent is when they say ‘it’s time.'”
For Nucci ‘it’s time’ meant starting the process of finding a living kidney donor in order to potentially avoid dialysis treatments and the further deterioration of his failing kidneys.
“I’m months away from dialysis,” said Nucci. “It’s inevitable, it’s going to come. But while dialysis is a life saver it’s not a good quality of life. The other problem is that if I have to go that route the longer I’m on dialysis waiting for a donor the less likely a kidney transplant will work or last.”
No stranger to rallying the troops in Eastie and citywide, Nucci, his wife, Peggy, and their three sons, John, Michael and Danny have launched a campaign in local Boston newspapers, on social media and through the relationships Nucci has cultivated over the years to find a living donor match.
Nucci’s wife and his boys have been quarterbacking the campaign to find a living donor as soon as possible. His three boys all tested positive for Polycystic Kidney Disease and were automatically eliminated as donors so they switched gears and began reaching out to other friends and family to find a match.
“I’m not social media savvy so my wife and the boys have been doing a tremendous job getting the word out on Facebook, making phone calls and reaching out to anyone and everyone,” said Nucci. “Finding a living donor is not the hard part and I have had dozens of people step up and get tested and for that I’m beyond grateful. A lot of people have already gone through the process of extensive testing at Mass General but for one reason or another they were eliminated. The problem isn’t so much finding someone who wants to donate it is finding a match. A direct donor has to be a direct blood type (Nucci is Type O) and Mass General is very diligent making sure that the donor is healthy and their kidneys are healthy.”
Nucci said waiting for a deceased donor could take seven to nine years and at his age the direct donor is really the only viable option.
“It’s a race against time,” he said. “I’m ready in terms of my own health. I am a healthy candidate for the transplant it’s just finding the match.”
In the meantime, his son, John, is running the Hyannis Sprint Triathlon to raise money and awareness for Polycystic Kidney Disease.
“In addition to raising money for this great cause, I’m also hoping to get the word out to as many potential donors as possible,” said John. “I know that it’s a huge ask, and it’s not something I would put out there if it wasn’t absolutely necessary, but we’re hoping that someone out there is willing and able to help him. I didn’t even know what a reasonable amount to target would be, and I’m honestly overwhelmed by the support. We’re raising money and getting the word out to so many more people and potential donors than my family and I possibly could’ve on our own, and it seriously means the world to all of us.”
Nucci’s wife Peggy has also been overwhelmed by the amount of support so far.
“I really want everyone to know how much we appreciate everyone’s willingness to help out with our efforts to find the matching kidney donor which John truly needs right now,” said Peggy.
For now, Nucci is trying not to think to much of what lies ahead and focus solely on coming out of this battle victorious, like he did so many times in his political career.
“I’m convinced I’m going to beat this,” said Nucci. “I have never won anything in my life without the help of my friends. My entire political life was spent relying on friends and supporters to win, and this is no different. In many ways it’s like another campaign and I’m going to need that support to win.”
If you or anyone you know is interested in becoming a donor, there is a quick and confidential online screening form that can be found at www.mghlivingdonors.org.
Dialysis Technology
From Medical Design & Outsourcing, By Danielle Kirsh
Intimidating medical devices can cause anxiety for patients — in the same way that people can experience “white coat hypertension” when seeing a doctor.
Devices that are smaller and more patient-centered could help patients feel better about their treatment and make them feel like they’re in charge, according to Leslie Trigg, CEO at Outset Medical (San Jose, Calif.).
“Because you are in control of it, it’s a different experience, even if it takes the same amount of time. It’s really about control. I think when you strip that all away, what really matters to us as human beings is the value we place on being independent and in control of our own destiny. And that’s really on a human level what the driving philosophy is behind Tablo,” Trigg said.
The Tablo is Outset Medical’s 3-foot-high kidney dialysis machine. It makes clean water, produces dialysate, takes blood pressure and delivers medication — all in one compact design. The machine is no taller than a desk and features a touchscreen interface meant to make treatment easier in clinics and hospitals. Its low-profile design is much smaller than other dialysis machines that require special filtration centers and large machines in the office.
“Tablo was launched in some of the existing 6,500 dialysis clinics [in the U.S.] to enable patients to do dialysis on their own. And this idea – we call in-center self-care – is really akin to self-serve dialysis. We kind of just borrowed on the concept of self-serve in retail,” Trigg said. “We are borrowing from that retail concept of self-care and bringing it into the dialysis setting because the system is so easy to use for the average patient. This is an opportunity for the patient to come in and set up Tablo on their own and manage their treatment.”
While Tablo is not currently available for home use, personalizing treatment and making it convenient helps patients feel at ease with their conditions. There are certain chemotherapy treatments that can be done at home, and the National Institutes of Health reports that it can improve patient outcomes and improve the quality of care.
Bringing the treatment closer to the patient is important since people are living longer and it provides a cost-effective solution to different treatments.
“People are living longer,” Trigg said. “If people are able to manage cancer as a chronic disease rather than an acute terminal episode, you have an opportunity – and obviously a responsibility – to make those sorts of chronic [therapies] that are maximumly cost-effective for patients and maximumly cost efficient for payers and providers.”
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