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Aeterna Zentaris: Phase 2 Data for Perifosine as Monotherapy for ... - PR Newswire (press release) PDF Print
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QUÉBEC CITY, June 18, 2012 /PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company") today announced that data from two Phase 2 trials in renal cell carcinoma (RCC) for the Company's oral anticancer drug, perifosine, have been published in the June 2012 issue of Cancer, a publication of the American Cancer Society. The article is titled, "Two Phase 2 Trials of the Novel Akt Inhibitor Perifosine in Patients with Advanced Renal Cell Carcinoma After Progression on Vascular Endothelial Growth Factor-Targeted Therapy", D.C. Cho, T.E. Hutson, W. Samlowski, P. Sportelli, B. Somer, P. Richards, J.A. Sosman, I. Puzanov, M.D. Michaelson, K.T. Flaherty, R.A. Figlin and N.J. Vogelzang. It outlines perifosine's activity as monotherapy in patients with advanced RCC, which was comparable to current second-line agents. Both objective tumor responses and prolonged disease stability were observed, and perifosine was well tolerated at the 100mg daily dose used in this trial. Finally, the authors conclude that this compound may be worthy of further investigation in this indication in combination with available therapies.

The Studies

Two single-arm, open-label Phase 2 trials (#228 and #231) were conducted to assess the efficacy and safety of perifosine as monotherapy in patients with advanced RCC who had failed on previous vascular endothelial growth factor (VEGF)-targeted therapy.

In the #228 trial, 24 patients with advanced RCC received oral perifosine (100 mg daily). The #231 trial enrolled 2 groups that received daily oral perifosine (100 mg daily): Group A comprised 32 patients who had received no prior mTOR inhibitor, and Group B comprised 18 patients who had received 1 prior mTOR inhibitor.

Results

In the #228 trial, 1 patient achieved a partial response and 11 patients had stable disease as their best response. The median progression-free survival was 14.2 weeks.

In the #231 trial, 5 patients achieved a partial response and 16 patients had stable disease as their best response. The median progression-free survival was 14 weeks both in patient with and without prior mTOR inhibitor therapy.

Overall, perifosine was well tolerated, and there were very few grade 3 and 4 events. The most common toxicities included nausea, diarrhea, musculoskeletal pain, and fatigue.

Juergen Engel, PhD, President and CEO of Aeterna Zentaris stated, "These data demonstrate perifosine's anticancer activity in monotherapy as well as its good tolerability. The authors' conclusion that perifosine may be worthy of further studies in combination therapy is in line with the strategy behind our current Phase 3 trial in multiple myeloma in which perifosine is combined with bortezomib and dexamethasone."

About Perifosine

Perifosine is a novel, oral anticancer drug that inhibits Akt activation in the phosphoinositide 3-kinase (PI3K) pathway. It is currently in a Phase 3 trial in multiple myeloma for which it has been granted orphan drug and Fast Track designations by the Food and Drug Administration, while also having received positive Scientific Advice and Orphan Medicinal Product designation from the European Medicines Agency. Furthermore, perifosine is in earlier-stage clinical development for other cancer indications. Rights for perifosine have been out licensed to Yakult Honsha Co. Ltd. for Japan, to Handok Pharmaceuticals Co. Ltd. for Korea and to Hikma Pharmaceuticals PLC for the Middle East and certain countries in North Africa. Aeterna Zentaris holds rights for the rest of the world.

About Aeterna Zentaris

Aeterna Zentaris is an oncology and endocrinology drug development company currently investigating treatments for various unmet medical needs. The Company's pipeline encompasses compounds at all stages of development, from drug discovery through to marketed products. For more information please visit www.aezsinc.com.

Forward-Looking Statements

This press release contains forward-looking statements made pursuant to the safe harbour provisions of the U.S. Securities Litigation Reform Act of 1995. Forward-looking statements involve known and unknown risks and uncertainties that could cause the Company's actual results to differ materially from those in the forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue R&D projects, the successful and timely completion of clinical studies, the risk that safety and efficacy data from any of our Phase 3 trials may not coincide with the data analyses from previously reported Phase 1 and/or Phase 2 clinical trials, the ability of the Company to take advantage of business opportunities in the pharmaceutical industry, uncertainties related to the regulatory process and general changes in economic conditions. Investors should consult the Company's quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties relating to forward-looking statements. Investors are cautioned not to rely on these forward-looking statements. The Company does not undertake to update these forward-looking statements. We disclaim any obligation to update any such factors or to publicly announce the result of any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, unless required to do so by a governmental authority or by applicable law.

SOURCE AETERNA ZENTARIS INC.

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Renal safety of tenofovir containing antiretroviral regimen in a ... - 7thSpace Interactive (press release) PDF Print
7thSpace Interactive (press release)
Renal safety of tenofovir containing antiretroviral regimen in a Singapore cohort - up-to-the-minute news and headlines. 7thSpace is a online portal covering

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Transplant recipient now advocate making available more lifesaving ... - Memphis Commercial Appeal PDF Print

Starting last July, Beverly Robertson, president of the National Civil Rights Museum, would wake up at 4:30 a.m. to get ready for her weekly kidney dialysis.

She would arrive at 5:15 a.m. at the dialysis center, where she sat in a treatment room while her blood flowed through a translucent plastic tube into a dialysis machine and newly clean blood flowed through another tube back into her body.

She repeated the routine for about nine months every Tuesday, Thursday and Saturday, until she had a kidney transplant in February.

She is now a self-appointed advocate for the Methodist University Hospital Transplant Unit. The hospital is attempting to gain support to receive a waiver from a federal regulatory decision to reduce the hospital's ability to receive organs from two organ procurement organizations and limit it to one that serves parts of West Tennessee and North Mississippi.

According to Dr. James Eason of Methodist, the change, which will go into effect in December, will reduce the pool of potential organ donors from about 7.5 million to 2 million.

It's a change that Robertson believes could take away opportunities for others to receive organ transplants, primarily for the liver, kidneys and pancreas.

"What it in essence means is that people are going to die," said Robertson, 60. "If they can't get a lifesaving organ, there are no options. The intervention is the transplant itself. I may not be here today if it were not for the ability to have a donor."

According to Eason, the average wait time for a kidney transplant in Memphis is about three years.

Robertson was an exception, but Eason said if she had not received the transplant, the probability of her receiving a new kidney after December would be drastically lowered.

"She was fortunate in that she got a close match fairly quickly," Eason said.

The Mid-South Transplant Foundation, however, supports the federal change, calling it a fairer method of distributing organs. Even with the smaller procurement area,

foundation officials say, there will be enough organs available for Memphis-area transplant patients.

Eason said along with diabetes, the top cause for kidney disease is hypertension or high blood pressure.

"I knew that the propensity for me to have it (high blood pressure) was pretty great," said Robertson. "What I didn't know was that high blood pressure can cause kidney damage."

Robertson visited her physician for a basic checkup and was sent the same day to a specialist, where she learned her high blood pressure was breaking down the blood vessels in her kidneys.

"I think sometimes we don't really understand how precious life is until our lives are threatened by something -- by some force -- that we may or may not have known about," Robertson said.

When speaking about her time on dialysis, Robertson's voice was steady and calm.

"It wasn't painful," she said. "It was just the time."

Her daughter, Adrienne Hines, worried that a transplant would be a long time in coming.

"That was my initial thought -- that she would be on the list for years and years and still undergo dialysis treatment," said Hines, a teacher at Hamilton Elementary.

Hines and her two younger brothers were tested to determine if they could be donors, but they don't share their mother's blood type, a crucial aspect of organ donation.

Eventually, Robertson's courage inspired the family to have faith that she would get a transplant.

"When she was on dialysis, she never had a day when she was depressed and wasn't herself," Hines said. "She was always bubbly and very energetic. Her spirit through it all was wonderful."

Though some dialysis patients are too weak to work, Robertson returned to work at the museum after her treatments.

She said very few of her employees knew of her condition because of her demeanor.

She wanted to keep the focus on the museum.

"It's very challenging. It can be stressful. But it's a calling -- the work that I do here," Robertson said. "So I don't view it as so much as a chore, but more so a real opportunity to be able to work here."

After the surgery, Robertson continued to see doctors to ensure that her body was accepting her new kidney. She took medication to prevent her immune system from attacking the new organ. She still continues medication to regulate her high blood pressure.

Robertson has spoken at various events about the regulatory controversy, including at the flag ceremony during National Kidney Awareness Week at Methodist and The Sisterhood Showcase banquet.

"She's a fireball," Eason said. "She's truly dedicated to trying to do what's right for this community."

She said she wants other patients on the organ donor waiting list to have the same opportunity she had.

"I feel incredibly blessed," Robertson said. "I want those same blessings to be in order for as many people as possible."

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30% hike in cost of dialysis in 6 months - Hindustan Times PDF Print
Sachin Bhutta, 35, has the same routine for the past four years, which includes half-a-day of dialysis thrice a week. A sales and marketing executive, Bhutta has no time, money or energy for any other activity, much like most chronic kidney disorder patients in the city.

Dialysis is a procedure one has to go through in case of acute kidney failure to remove waste and unwanted water from the body. In the past six months, the cost of this procedure has gone up by 30% and nearly doubled in the past one year, doctors said.

“I spend around Rs14,000 on dialysis every month and another Rs10,000 for injections and medicines. I spend most of my salary on this and am unable to contribute at home,” said Bhutta, who was detected with chronic kidney disorder in 2005 when he was 28 years old and decided against getting married.

Until last year, actual cost of dialysis was around Rs500 and around Rs700 until six months ago. But owing to the increase in prices of chemicals used in the procedure, instruments and electricity bills, it now costs Rs1,000 per dialysis session,” said Dr Umesh Khanna, nephrologist and head of Mumbai Kidney Foundation.

He said while the civic body provided only 25 dialysis machines, private sector has around 700 machines. The Brihanmumbai Municipal Corporation has announced 51 more dialysis machines.

At present there are around 15,000 patients on dialysis in the city. “We are encouraging public private partnership where we would provide the space and NGOs would run those centres,” said Manisha Mhaiskar, additional commissioner, BMC. At municipal hospitals, one dialysis session costs Rs. 400.

“Mumbai has the highest number of dialysis centres run by non-profit organisations in India, which offer dialysis for as little as Rs300 to Rs. 500. They cannot accommodate all the needy patients,” Khanna said.

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Sunday Mail legend Rita Madhok's renal charity set to hit £1m as 25 ... - Scottish Daily Record PDF Print
Rita Madhok with husband Rajan

HER enthusiasm, passion and laughter drew even strangers to her…Rita Madhok loved people and they returned her warmth in spades.

Today, that same energy and zest which made Rita – better known as Sunday Mail astrologer Darlinda – so special has kept her friends and family working tirelessly for the charity launched in her name.

As the fundraising for renal research approaches the £1million mark, they can be satisfied that she would have been a very happy woman.

Her husband Rajan said: “There are so many times when we have said to ourselves, ‘It’s almost as if she is still with us.’

“She would have been absolutely thrilled by how much money we have raised in her name and she was always very grateful for anything anybody did to help, no matter how small.

“She was driven to care. Even when she was ill and I would tell her to slow down, she would say, ‘There are other people who are not as lucky as we are and don’t have the same support.’

“She was determined to do something to help them.”

As psychic and clairvoyant Darlinda, for 16 years Rita was known across ­Scotland as the Sunday Mail’s much-loved astrologer.

She died in 1995 after suffering diabetes which led to kidney failure.

For years she was on dialysis, a debilitating and time-consuming treatment but – transplant apart – the only way to stay live.

Rajan, a doctor, said: “When you are in renal failure, you are just totally nauseated, feeling sick and ghastly all the time.

“She would go up to the Western Infirmary in Glasgow for her ­appointments feeling awful.

“In 1987 her life was transformed by a transplant and we had eight to nine excellent years.

“It was a good time and it made such a difference to her life, from one day being tied to the dialysis to the next when she was up and about again.

“It is really life-changing but she did not forget what it had been like before and she wanted to do something to improve the facilities available to patients and make it a more comfortable
environment – less clinical.”

The result was the establishment of Darlinda’s Charity for Renal Research – which began with her seven-month-old nephew, who did a sponsored crawl across the living room floor.

It took off when her good friend Sir Hugh Fraser waded in with a “substantial” cheque and the annual balls began. The events have been the charity’s main ­fundraiser over the years.

One of the first things bought by Darlinda and friends was the kidney unit’s ultrasound machine. Until then, renal patients had to trek to the basement of the hospital to get an ultrasound of their kidneys at X-ray.

Rajan said: “Lots of people knew Rita and there were humble beginnings – people just doing little things – then the realisation that we were serious and then Sir Hugh came on board as our first major benefactor.”

By the time of her death in 1995, Rita had raised almost £200,000 by her ceaseless fundraising efforts.

Rajan said: “Transplanted kidneys have a limited lifespan and it was a combination of things arising at the same time – vascular complications – but the kidney basically began to fail.”

Rita went out as she had lived her life – with her family and friends around her and a bottle of champagne in a bucket by the bedside.

Rajan said: “We had a very good life and were able to do lots of things. The charity formed an important part of that because we were so grateful for the gift of life and Rita felt it was so important to ­reciprocate and give something back to people who were not so fortunate.”

Over the years the charity have supplied immediate help to kidney patients, purchasing home ­dialysis machines and setting up a patient comfort fund to buy little things that would make life better on the ward.

They tend to support initial – usually unsupported – research, helping scientists secure further funding. They have funded labs and stem cell research and studies into the links between heart problems and renal failure.

They have even funded the Transplant Games and are the biggest single supporter of the kidney unit at the Western Infirmary.

This year marks the 25th anniversary of the charity and is, appropriately, the year in which they look like bursting through the £1million mark. The total raised to date is around £950,000.

Next Saturday endurance runner Charlie Gordon will add to that total by trying to complete 95 miles of the West Highland Way in less than 35 hours.

On November 10 the annual Dynasty Ball should take the total through the barrier.

Rajan said: “It is going to be a wonderful feeling.

“I hope we can share with all the friends who have helped us get to this moment.”

 

CHARLIE GORDON

Endurance runner Charlie Gordon is going a long way to edge Darlinda’s charity towards the magic million.

He is taking part in the annual West Highland Way race, which sets runners off along the 95 miles of the walkway with only 35 hours in which to complete it.

The 43-year-old has done part of the route before as a support runner, motivating other runners to the finish line.

Charlie Gordon

But this year, for the first time, he will take on the whole route and try to finish it in 28 hours.

For that, Charlie, a commercial manager with structural steel firm J D Pierce, hopes to hand over at least £2000.

But it will be no picnic – and the weather as well as the midges can be crucial.

Charlie said: “It’s easy to lose two hours at a stop having a cup of soup, changing your shoes or putting on midge spray.

“Then there are the sleep and endurance issues and keeping fuelled up.

“You’ve got to get calories into you and anything from 150 to 200 calories an hour is challenging. The trick is to eat little and often and to keep moving forward.”

The race starts on Saturday at 1am in Milngavie, near Glasgow, and has strict protocols.

Runners must have completed at least one 50-mile endurance race before and have support runners to race alongside at different points on the route as well as a motorised back-up team.

If all goes well, Charlie will cross the finishing line in the early hours of Sunday morning – and Darlinda’s charity will be £2000 richer.

He said: “The challenge in these things is if the wheels come off and, between your team and your own
mental strength, it’s getting the wheels back on.

“There are lots of different ways you can come a cropper but I want to finish well for the charity.

“At the end of the day, that’s what’s important.”

 

LARURA RITCHIE

No one knows better than Laura Ritchie just how vital Darlinda’s charity is.

It is there for big things such as research projects – and for little things such as DVD players when you are spending the long, hard hours in dialysis.

When she was 21, Laura was struck down by vasculitis, an auto-immune disorder that attacks the blood vessels in any part of the body – in Laura’s case, it was her kidneys.

Laura Ritchie with husband Callum

She was put on dialysis but was so ill her parents and boyfriend were told she might not last the night.

Laura fought back and over the next two weeks was given seven blood transfusions – but her life changed dramatically.

She had been studying in Madrid but had to come home as she needed dialysis three times a week for five hours. The break in her studies meant she had to start the year again the following September.

Laura said: “It was a big life shock and change.”

In the meantime, a family friend had come forward and was found to be a match for Laura, which resulted in a frantic time.

She said: “I sat my last exam on Monday, got my transplant on the Friday and graduated five weeks later. It was amazing.”

Laura, an accountant with Tennent’s, was drafted in to explain to people how important funding is for the unit at the Western Infirmary.

To date, her transplant is good and two years ago she married childhood sweetheart Callum.

She said: “No one knows how long a kidney will last. That’s what makes Darlinda’s so important – they work all the time to help people like me.”

 

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