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ACADIA Pharmaceuticals and Neuren Pharmaceuticals Announce Rare Pediatric Disease Designation for Trofinetide for the Treatment of Rett Syndrome
5:50 PM ET 3/3/20 | BusinessWire
With No Approved Treatments in Rett Syndrome, FDA Decision Highlights Significant Unmet Need
SAN DIEGO & MELBOURNE, Australia--(BUSINESS WIRE)--March 03, 2020--
ACADIA Pharmaceuticals Inc. (Nasdaq: ACAD) and Neuren Pharmaceuticals Limited (ASX: NEU) announced today that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease (RPD) designation to trofinetide for the treatment of Rett syndrome, a serious and rare neurological disorder. Upon FDA approval of a product with RPD designation, the sponsor may be eligible to receive a Priority Review Voucher, which can be used to obtain FDA review of a New Drug Application for another product in an expedited period of six months.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200303006075/en/
"We are pleased that the FDA has recognized the unmet need currently experienced by Rett patients and their families and our goal is to bring a treatment option forward as soon as possible," said Serge Stankovic, M.D., M.S.P.H., ACADIA's President. "This is an encouraging step forward as we continue to enroll patients in our Phase 3 LAVENDER study with results expected in 2021."
RPD designation is granted by the FDA in the case of serious or life-threatening diseases affecting fewer than 200,000 people in the U.S. and primarily in individuals 18 years of age and younger. Trofinetide was previously granted Fast Track Status and Orphan Drug Designation for Rett syndrome in the U.S. and Orphan Drug Designation for Rett syndrome in Europe.
About Rett Syndrome
Rett syndrome is a debilitating neurological disorder that occurs primarily in females following apparently normal development for the first six months of life. Rett syndrome has been most often misdiagnosed as autism, cerebral palsy, or non-specific developmental delay. Rett syndrome is caused by mutations on the X chromosome on a gene called MECP2. There are more than 200 different mutations found on the MECP2 gene that interfere with its ability to generate a normal gene product.
Rett syndrome occurs worldwide in approximately one of every 10,000 to 15,000 female births and in the United States impacts 6,000 to 9,000 patients. Rett syndrome causes problems in brain function that are responsible for cognitive, sensory, emotional, motor and autonomic function. Typically, with symptoms presenting between six to 18 months of age, patients experience a period of rapid decline with loss of purposeful hand use and spoken communication and inability to independently conduct activities of daily living. Symptoms also include seizures, disorganized breathing patterns, an abnormal side-to-side curvature of the spine (scoliosis), and sleep disturbances. Currently, there are no FDA-approved medicines for the treatment of Rett syndrome.
About Trofinetide
Trofinetide is an investigational drug. It is a novel synthetic analog of the amino--terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function. In the central nervous system, IGF-1 is produced by both of the major types of brain cells -- neurons and glia. IGF-1 in the brain is critical for both normal development and for response to injury and disease. Trofinetide has been granted Fast Track Status and Rare Pediatric Disease Designation for the treatment of Rett Syndrome in the U.S. and Orphan Drug Designation in the U.S. and Europe for both Rett syndrome and Fragile X syndrome.
The Phase 3 trofinetide clinical program includes LAVENDER, a 12-week, double-blind, placebo-controlled study, and LILAC, an open-label, long-term extension study. The Phase 3 clinical program is progressing as planned with 11 study sites recruiting and more sites expected in the future.
About ACADIA Pharmaceuticals
ACADIA is a biopharmaceutical company focused on the development and commercialization of innovative medicines to address unmet medical needs in central nervous system disorders. ACADIA has developed and commercialized the first and only medicine approved for the treatment of hallucinations and delusions associated with Parkinson's disease psychosis. ACADIA also has ongoing clinical development efforts in additional areas with significant unmet need, including dementia-related psychosis, the negative symptoms of schizophrenia, major depressive disorder, and Rett syndrome. This press release and further information about ACADIA can be found at www.acadia-pharm.com.
In 2018, ACADIA entered into an exclusive North American license agreement with Neuren for the development and commercialization of trofinetide for Rett syndrome and other indications. Under the terms of the license agreement between ACADIA and Neuren, Neuren is eligible to receive one third of the market value of any Rare Pediatric Disease Priority Review Voucher, if awarded by the U.S. FDA upon approval of a New Drug Application for trofinetide.
About Neuren Pharmaceuticals
Neuren Pharmaceuticals Limited (Neuren) is a biopharmaceutical company developing new therapies for brain injury, neurodevelopmental and neurodegenerative disorders. Neuren has completed Phase 2 development of trofinetide for Rett syndrome and has completed a Phase 2 clinical trial in Fragile X syndrome.
Forward-Looking Statements
Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements include but are not limited to statements regarding the Phase 3 clinical trial evaluating trofinetide; the likelihood of success of such clinical trial; the prospects for FDA approval of trofinetide for Rett syndrome and other indications; and the success of any efforts to commercialize trofinetide in North America. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks and uncertainties inherent in drug discovery, development, approval and commercialization. For a discussion of these and other factors, please refer to ACADIA's annual report on Form 10-K for the year ended December 31, 2019 as well as ACADIA's subsequent filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All forward-looking statements are qualified in their entirety by this cautionary statement and ACADIA undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof, except as required by law.
This ASX-announcement contains forward-looking statements that are subject to risks and uncertainties. Such statements involve known and unknown risks and important factors that may cause the actual results, performance or achievements of Neuren to be materially different from the statements in this announcement.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200303006075/en/
CONTACT: ACADIA Pharmaceuticals Inc.
Media Contact:
Stephanie Fagan
(858) 212-0534
media@acadia-pharm.com
or
Investor Contact:
Mark Johnson, CFA
(858) 261-2771
ir@acadia-pharm.com
or
Neuren Pharmaceuticals Limited
Contact:
Jon Pilcher
Chief Financial Officer & Company Secretary
+61 438 422 271
jpilcher@neurenpharma.com
SOURCE: ACADIA Pharmaceuticals Inc.
Copyright Business Wire 2020
> Dow Jones Newswires
March 03, 2020 17:50 ET (22:50 GMT)
Hagen should not be donating $5k to breast cancer, when POTN is bankrupt. Also, if he is going to donate, why not donate to the prostate/testicle cancer foundation? It's such a gyno-centric society, aren't men important too? The other day, I saw a pink trash can, meaning it's supporting breast cancer, so why not have a blue trash can in support of balls and testie/prostate cancer?
Uh oh, ATRS goes splat....
Well, within a couple of days, it went up to $60 and has been down ever since, lol.
Even if Tlando was FDA approved in 2020, on the same day of approval, there would probably be a dilutive offering, thereby torpedoing the share price.
You had better hope the you are raptured out, before experiencing tremendous loses in Lipocine.
Customer Service would not know, or tell you if POTN is going out of business.
I sort of understand POTN wanting to get out of the vaping business, due to lawsuits from vaping deaths.
If POTN takes melatonin out of the if products, like the CDB gummies, I doubt they would cause a relaxing/tired feeling.
I am surprised that POTN will be taking melatonin out of their products.
GoPro's IPO was $60 per share. Obviously, it was a big joke, that the so called analysts got wrong, gee talk about fake news!
Ummm no,
Due to all the FDA rejections the steam has been taken out of Tlando's potential boost in share price.
Also, the share price might tank after Tlando approval, due to Lipocine not having the money to market Tlando.
Learn from the history of rejection, rejection, rejection, then approval, LOL!
I just got off the phone with my friend Rupert and here's what he told me...
1. POTN is at its bottom.
2. Now is the time to average down on POTN.
3. The FDA has a new head (thanks to President Trump) that is pro CBD and regulation issues will be resolved.
4. The government will win the case against the 3 fraudsters and the POTN shares will be disgorged, returned to POTN and retired.
6. The caveat emptor symbol will be removed.
7. POTN won't do a reverse split.
8. The dip in POTN sales is over.
9. POTN sales will increase from here.
10. POTN will enter into MJ sales.
11. The CEO will have a flurry of news releases.
12. More investments will be made into POTN.
13. The POTN stock price will rise.
Rupert has a nose for stocks, he is underwater at this time, but Rupert remains confident. All of Rupert's other investments are in the green, from when he took a position. Rupert has a knack for getting in early.
Dr.Damaj has torpedo'd your stock price and company!
Last one out, please turn out the lights.
ACAD seems to be doing ok so far, during the market correction/corona virus attack.
I wonder if ACAD will remain around $40 per share or tank back down to $17 or so.
I dare anyone here to average down, in the midst of a reverse split and the bankruptcy of POTN.
With all of the Tlando rejections, even if the FDA were to approve Tlando, I doubt the share price would rise much, due to all of the hype being taken out of the approval, due to the multiple rejections.
I say this, because I've seen it happen before, with other drugs being approved after multiple rejections and the share price not rising.
Therefore, an increase in share price would probably only occur due to high, Tlando sales, which it's hard to get a new drug launched and off the ground.
In order to launch Tlando, I would expect a reverse split, or some drastic type of financing, to torpedo the increase of LPCN's share price, for years to come.
Unless, there is a buy out of Lipocine, but I don't think there is much if any interest in Lipocine.
Due to society being so gyno-centric, Lipocine would probably do better to have invented a drug, that turns men into women, unfortunately.
Well, my pps is .12 and I'd like to get out at a break even asap, obviously without an r/s or bankruptcy, but I just don't see it happening.
Oh the pain! My hole has been cored out on this one. I got in at around 4.+
Oh the pain! My hole has been cored out on this one. I got in at around 4.+
LOL, even if the RSI of 34 was the actual PPS, I would still not even recoup 50% of my INNV losses.
We should have a PPS of at least $60 per share, w/o an R/S and then I could leave you and this board behind.
Do not forget, Dr.Bassam Damaj from INNV ruins any company he's a part of, so AYTU is toast.
I just received an email from Diamond for 80% until the end of the month.
This is a last ditch, desperate move to keep from bankruptcy.
However, it won't prevent the r/s, more dilution, or going out of business.
Still a dormant pharma company, how dumb! When will this POS be revere shell merged into a new company that want's to go public, what a damned joke!!!
Mulally, you asked "Why can you not buy more?" The answer is because Dr.Damage is a parasite, who is now ruining AYTU.
Anyone thinking of averaging down, better guess again! You will lose every penny, due to the bankruptcy.
Well, long story short...
Business model has changed several times with multiple gimmicks.
Business was born from shady and dishonest used car salesmen.
The 3 or more pump and dump crooks heading to trial.
Constant and extreme, massive dilution.
Several missed guidance's.
FDA and big pharma blocking CDB/THC.
Shady India auditing.
Missed up-listing.
Caveat Emptor symbol.
Low trading volume.
Mismanagement, due to a variety of reasons, magazine, slushi vans, bit-coin, etc.
Lower and lower sales.
Much big and much better managed competitors.
Charles Vaccaro emptied the sewage-poop from his latest Yacht, into the Florida bay and got busted.
The latest product will be edible hemp diapers that you eat after the baby has filled them up.
Hi Richard, POTN is in the process of going bankrupt for a variety of reasons, too many for me to list here.
Uh oh, get ready for the 3rd or 4th failure sometime in 2021 for the re submission of TLando...
https://seekingalpha.com/news/3544840-lipocine-soars-on-plan-to-resubmit-nda-for-testosterone-drug
Lipocine (LPCN +38.3%) jumps to three-month highs after saying the Food and Drug Administration indicated the company's approach to addressing the single remaining deficiency in its New Drug Application for oral testosterone product candidate Tlando appears to be a reasonable path forward.
SALT LAKE CITY, Feb. 24, 2020 /PRNewswire/ -- Lipocine Inc. (LPCN), a clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, today announced the results of its Post Action meeting with the U.S. Food and Drug Administration ("FDA') regarding its New Drug Application ("NDA") for TLANDO™, the Company's oral testosterone product candidate for testosterone replacement therapy ("TRT") in adult males for conditions associated with a deficiency of endogenous testosterone, also known as hypogonadism. Based on the Post Action meeting and written feedback, the FDA indicated Lipocine's approach to addressing the single remaining deficiency through the reanalysis of existing data in accordance with FDA feedback appears to be a reasonable path forward. The FDA requested that the information generated by the reanalysis be submitted as part of an NDA resubmission with a six-month Prescription Drug User Fee Act ("PDUFA") clock.
"We are committed to working closely with the FDA towards the goal of achieving approval of TLANDO, our fixed-dose oral TRT," said Dr. Mahesh Patel, Chairman, President and Chief Executive Officer of Lipocine. "We plan to submit the TLANDO NDA in the first quarter of 2020."
LOL, Fluticare has been out of stock, because Dr.Damage had to be paid, screw the shareholders...
I understand what you're saying, but for AYTU to stay on the Nasdaq, they will probably need to do yet another revers split.
I doubt they will bring back Fluticare (maybe a small stock at around Winter time.)
Redundant products, dumb snake oil stuff.
God damn Dr.Bassam Damaj to Hell, for lying and ripping us off.
Dr.Damage is still on the management BOD, so he's ruining AYTU with his disease.
Noode12, from here, we go to bankruptcy. Even if the corrupt FDA clarified CBD rules, POTN is so far gone already, it wouldn't matter.
I'm not surprised ACAD is going down before Q4 is announced.
Mulally, in most cases you would be correct that I still believe, but I honestly don't believe. The only reasons that I haven't sold is because I've already lost so much, what difference does it make if I loose just a little bit more?
Also, I was too embarrassed to show my tax lady, the extreme losses if I had sold.
Lol, I doubt it. We've got 2 losers here put together. It's 100% loss for me.
Hi, in what why does the low volume look interesting?
What are you hinting at?
I'm just trying to learn as much as I can from the view of others, so let me know.
Thank you.
INNV is already cauing AYTU to go down and this is just the beginning of AYTU's downfall.
Remember...
Bassam Damage is still in management and his toxic spread of causing disease to companies is occurring, even as I type this.
Bassam Damaj repeatedly and knowing lied to INNV investor and ripped INNV investors off.
I'm not talking about small downward trends, I'm talking about 100% losses for INNV investors and soon to be 100% losses for AYTU investors as well...
But, don't worry Bassam will still be able to eat lots of lobster, steak and caviar, as well as sail in his yacht.
AYTU has had multiple revers splits, what a joke!
INNV has had a reverse split, what a joke!
AYTU and INNV are both craptacular businesses...
Look for AYTU to be kicked off the Nasdaq...
Look for more dilution...
Look for another reverse split...
Look for bankruptcy...
Every penny invested in AYTU and INNV will be lost...
luvgrowth indicated that this would go back to $3.50 to fill the gap and low and behold, he was correct.
Re: "POTN$ 0.10 COMING SOON! NO R/S."
What makes you say this? What makes you confident of that?
Jack, most everytime you post you start with a "Hey, Bulba..."
Who is Bulba?
Lol, is that acronym for something?
Well, Innovus was a scam from the beginning, anyone who founds a company on sex lube, premature cum lube, sex enhancement lube is a damned joke!
Oh ya, I'm Innovus and I have a volume-contract with all the major porn producers, to send them 50 gallon drums of lube!
Uh Brazman, POTN will be out of business very soon.