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Cystic fibrosis – Symptoms and causes – Mayo Clinic

Posted: February 29, 2020 at 11:03 pm

Overview

Cystic fibrosis (CF) is an inherited disorder that causes severe damage to the lungs, digestive system and other organs in the body.

Cystic fibrosis affects the cells that produce mucus, sweat and digestive juices. These secreted fluids are normally thin and slippery. But in people with CF, a defective gene causes the secretions to become sticky and thick. Instead of acting as lubricants, the secretions plug up tubes, ducts and passageways, especially in the lungs and pancreas.

Although cystic fibrosis is progressive and requires daily care, people with CF are usually able to attend school and work. They often have a better quality of life than people with CF had in previous decades. Improvements in screening and treatments mean that people with CF now may live into their mid- to late 30s or 40s, and some are living into their 50s.

In the U.S., because of newborn screening, cystic fibrosis can be diagnosed within the first month of life, before symptoms develop. But people born before newborn screening became available may not be diagnosed until the signs and symptoms of CF show up.

Cystic fibrosis signs and symptoms vary, depending on the severity of the disease. Even in the same person, symptoms may worsen or improve as time passes. Some people may not experience symptoms until their teenage years or adulthood. People who are not diagnosed until adulthood usually have milder disease and are more likely to have atypical symptoms, such as recurring bouts of an inflamed pancreas (pancreatitis), infertility and recurring pneumonia.

People with cystic fibrosis have a higher than normal level of salt in their sweat. Parents often can taste the salt when they kiss their children. Most of the other signs and symptoms of CF affect the respiratory system and digestive system.

The thick and sticky mucus associated with cystic fibrosis clogs the tubes that carry air in and out of your lungs. This can cause signs and symptoms such as:

The thick mucus can also block tubes that carry digestive enzymes from your pancreas to your small intestine. Without these digestive enzymes, your intestines aren't able to completely absorb the nutrients in the food you eat. The result is often:

If you or your child has symptoms of cystic fibrosis or if someone in your family has CF talk with your doctor about testing for the disease. Consult a physician who is knowledgeable about CF.

Cystic fibrosis requires consistent, regular follow-up with your doctor, at least every three months. Contact you doctor if you experience new or worsening symptoms, such as more mucus than usual or a change in the mucus color, lack of energy, weight loss, or severe constipation.

Seek immediate medical care if you're coughing up blood, have chest pain or difficulty breathing, or have severe stomach pain and distention.

In cystic fibrosis, a defect (mutation) in a gene the cystic fibrosis transmembrane conductance regulator (CFTR) gene changes a protein that regulates the movement of salt in and out of cells. The result is thick, sticky mucus in the respiratory, digestive and reproductive systems, as well as increased salt in sweat.

Many different defects can occur in the gene. The type of gene mutation is associated with the severity of the condition.

Children need to inherit one copy of the gene from each parent in order to have the disease. If children inherit only one copy, they won't develop cystic fibrosis. However, they will be carriers and could pass the gene to their own children.

Because cystic fibrosis is an inherited disorder, it runs in families, so family history is a risk factor. Although CF occurs in all races, it's most common in white people of Northern European ancestry.

Complications of cystic fibrosis can affect the respiratory, digestive and reproductive systems, as well as other organs.

If you or your partner has close relatives with cystic fibrosis, you both may choose to have genetic testing before having children. The test, which is performed in a lab on a sample of blood, can help determine your risk of having a child with CF.

If you're already pregnant and the genetic test shows that your baby may be at risk of cystic fibrosis, your doctor can conduct additional tests on your developing child.

Genetic testing isn't for everyone. Before you decide to be tested, you should talk to a genetic counselor about the psychological impact the test results might carry.

Feb. 04, 2020

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LAFC vs. Inter Miami CF most in-demand opening game ticket in at least 10 years – MLSsoccer.com

Posted: at 11:03 pm

The season-opening bout between Inter Miami CF and LAFC at Banc of California Stadium on Sunday (5:30 pm ET | ESPN, ESPN Deportes, MLS LIVE on DAZN in Canada)is a sexy matchup for a lot of reasons.

On one side, you've got an LAFC team coming off arguably the most dominant single season in MLS history a torrid 2019 campaign that saw them cruise to the Supporters' Shield while racking up 72 points and post a ridiculous +48 goal-differential (both league records)while also boasting the league's MVP in Carlos Vela.

On the other, you've got Inter Miami playing theirfirst-ever MLS match, a long-awaitedculmination of an expansion project years in the making, with a roster that looks set to have them competitive from the jump, including a Mexican star of theirown in Rodolfo Pizarro.

All of that makes it arguably the most anticipated fixture of the league's opening weekend, and it's reflected in the numbers.

According to a Thursday story in the Miami Herald, tickets for the match are re-selling for an average price of $149 the highest number for an MLS opener since SeatGeek started tracking the secondary market in 2010. The next-closest total for this year's opening weekend is Nashville SC's MLS debut against Atlanta United on Saturday at $84.

If you needed any more evidence as to the hype surrounding this one, that's as good as any. Now, to see how it actually plays out on the field.

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Local third grader reads his newly published book about living with CF to classmates – RochesterFirst

Posted: at 11:03 pm

Posted: Feb 28, 2020 / 06:44 PM EST / Updated: Feb 28, 2020 / 07:35 PM EST

ROCHESTER, N.Y. (WROC) Students at School No. 43 in Rochester gathered to support their classmate living with cystic fibrosis, or CF.

At Fridays Purple Shirt Day assembly, third grader Trent Judkins read aloud his own recently published book.

8-year-old Judkins is living with cystic fibrosis. The hereditary disease affects the lungs and the digestive system. His book that he wrote and illustrated explains what its like to have the disease.

Trents father, Trent Judkins Sr., helped to organize the assembly.

Today were just looking to kinda raise awareness. Really just help families that are dealing with CF. Kinda put it out there let it be known that its known in our community. Just kind of give everyone a visual on exactly what happens in your day to day life and what goes on with CF and cystic fibrosis, said Trent Judkins Sr.

Trents book is called A Boy with CF: His Story of Acceptance and Endurance. Trent and his family also started a foundation for CF called TJAE CF Foundation, which stands for The Journey of Acceptance and Endurance.

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Proteostasis Therapeutics and CF Europe Announce Completion of Patient Enrollment for CHOICES, the First-Ever Personalized Medicine-Based Study in…

Posted: at 11:03 pm

Ex Vivo Testing Phase Underway with Tissue Samples Collected from More than 500 Adult CF Patients; Enrollment Target Exceeded

Clinical Testing Phase to Begin in 2H 2020

Company Expects CHOICES to Potentially Serve as the Basis for an MAA in 2021

BOSTON, Feb. 24, 2020 /PRNewswire/ --Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and CF Europe, the federation of 48 national CF Associations in Europe, today announced the completion of enrollment of 502 patients with CF for HIT-CF, a European-based initiative that is paving the path to personalized medicine through the CHOICES clinical trial. CHOICES will test PTI drug combinations in an ex vivo study and then in a clinical trial to assess the predictability of the organoid assay for clinical benefit.

For the ex vivo portion, organoids derived from tissue samples provided by patients enrolled in the study are evaluated for responsiveness to investigational CFTR modulators, including Proteostasis' CFTR potentiator, corrector and amplifier, dirocaftor (DIR), posenacaftor (POS) and nesolicaftor (NES), respectively. Based on an individual's organoid response, patients will be invited to progress to the next portion of the study which is a placebo controlled, double blind, crossover study known as the CHOICES trial (Crossover trial based on Human Organoid Individual response in CF - Efficacy Study).

The results from CHOICES may serve as the basis for a potential Marketing Authorization Application with the European Medicines Agency (EMA) in 2021 through a novel regulatory pathway. This strategic initiative is led by the HIT-CF consortium, funded through the European Commission's Horizon 2020 program. The CHOICES clinical study is part of PTI's broader clinical development strategy for its CFTR modulator candidates that also includes the MORE trial in CF subjects with the most common F508del homozygous genotype.

"The enrollment of more than 500 patients across Europe in the first phase of the HIT-CF project is a testament to the strategic imperative this program holds for both the patient and treatment community," said Geoffrey Gilmartin, M.D., M.M.Sc., Chief Medical Officer of Proteostasis Therapeutics. "With the successful translation of activity from organoids to patients, this study has the potential to usher in a personalized medicine approach to CF. This approach would begin with patients who have less common mutations, but could ultimately serve the broader CF community by delivering personalized treatment choices that maximize benefit based on each patient's responsiveness to therapy."

"We are excited that Proteostasis is participating in the HIT-CF project and supporting our efforts to bring CF treatment to more people across Europe," said Jacquelien Noordhoek, President of CF Europe and representative of the Netherlands Cystic Fibrosis Society (NCFS). "Enrolled individuals are a portion of the approximately 2,300 adults in the European patient registry who are not eligible for any currently approved modulator due to their genotype and the HIT-CF project represents the only option to explore potential benefit of disease modifying drugs for this group. Putting patients with CF first is our highest priority. We are looking forward to continuing our partnerships and providing Europeans with CF the best possible care."

About Organoids

Organoids are cell cultures that grow in a culture dish with properties similar to those of the organ from which they are derived. Because organoids are made from stem cells, they contain the same mutations as the person from whom the biopsies are derived. Investigational drugs which target the basic defect of CF can be used in an organoid system to evaluate rare mutations where the drugs may have a positive effect.

Unlikein vitrosystems such as human bronchial epithelial (HBE) cells, which are derived from lungs that have been removed from CF patients, or the engineered rat-derived FRT cell line (which has had false positive clinical results), rectal organoids are cultured from tissues obtained through a minimally invasive and painless procedure from donors who then become eligible to participate in a clinical study. Organoids can provide valuable insights for donors, including their likelihood of achieving improvements in pulmonary function and reductions in sweat chloride concentration with CFTR modulators based on theex vivoresponse to those drugsi.

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About HIT-CF Europe

HIT-CF Europe is a research project which aims to provide better treatment and better lives for people with cystic fibrosis (CF) and rare mutations. To achieve this, drug candidates are first tested on patient-derived organoids in qualified laboratories acrossEurope. Subsequently, based on the measured signal in the organoids, a smaller group of patients will be invited to participate in clinical trials with investigational molecules from participating pharmaceutical companies.

All participating centers are part of theEuropean Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN). The project has received funding from theEuropean Union'sHorizon 2020 research and innovation program under grant agreement number 755021. For more information, visitwww.hitcf.org.

About Proteostasis Therapeutics, Inc.

Proteostasis Therapeutics, Inc.is a clinical stage biopharmaceutical company developing small molecule therapeutics to treat cystic fibrosis and other diseases caused by dysfunctional protein processing. Headquartered inBoston, MA, theProteostasis Therapeuticsteam focuses on identifying therapies that restore protein function. For more information, visitwww.proteostasis.com.

Safe Harbor

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including but not limited to statements regarding the potential of PTI drug combinations, expectations regarding ex vivotesting of our proprietary combinations in organoids and clinical evaluation in CF patients, the expected timing for enrollment, completion and reporting of results of our CHOICES Phase 3 clinical trial, our commitment to expanding available therapeutic options for CF patients and the intended goals of the CHOICES trial and the ability to serve as a potential basis for future marketing approval. Words such as "aim," "may," "will," "expect," "anticipate," "estimate," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved. Such risks and uncertainties include, without limitation, the potential of our proprietary combination therapies for the treatment of CF, the potential benefit of our proprietary combination therapies to patients, expected completion of our clinical studies and cohorts for our clinical programs, initiation of a pivotal or registrational study, the possibility final or future results from our drug candidate trials (including, without limitation, longer duration studies) do not achieve positive results or are materially and negatively different from or not indicative of the preliminary results reported by the Company (noting that these results are based on a small number of patients and small data set), uncertainties inherent in the execution and completion of clinical trials (including, without limitation, the possibility that FDA or other regulatory agency comments delay, change or do not permit trial commencement, or intended label, or the FDA or other regulatory agency requires us to run cohorts sequentially or conduct additional cohorts or pre-clinical or clinical studies), in the enrollment of CF patients in our clinical trials in a competitive clinical environment, in the timing of availability of trial data, in the results of the clinical trials, in possible adverse events from our trials, in the actions of regulatory agencies, in the endorsement, if any, by therapeutic development arms of CF patient advocacy groups (and the maintenance thereof). For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. We assume no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, unless required by law.

CONTACTS:

Investors:David Pitts / Claudia StyslingerArgot Partners212.600.1902david@argotpartners.com/ claudia@argotpartners.com

Media:David RosenArgot Partners212.600.1902david.rosen@argotpartners.com

HIT-CF Project Coordination:HIT-CF ConsortiumUniversity Medical Centre Utrecht (The Netherlands)HITCF@umcutrecht.nl

i Berkers et al, Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis Cell Reports 26, 17011708,February 12, 2019

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SOURCE Proteostasis Therapeutics, Inc.

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Inter Miami CF unveil new 2020 MLS home jersey: See what the expansion club will wear – MLSsoccer.com

Posted: at 11:03 pm

Inter Miami CF wont play their first home game in Major League Soccer until March 14 against the LA Galaxy (2:30 pm ET | FOX, MLS LIVE on DAZN, FOX Sports App, FOX Deportes), but we now know what jersey theyll wear for their Fort Lauderdale stadium debut.

The 2020 expansion club unveiled their home kit on Monday, providing a contrast to the clubs black away kit, which was unveiled in early February. The home one is white, with various elements touching on Inter Miamis black and pink colorway.

The collar features a singular pink button, while the jerseys inside neck tape has the phrase Freedom to Dream spelled out in all caps in black lettering. The same pink colorway is used on the three shoulders stripes, a hallmark of adidas jerseys.

The clubs largely-black badge provides acontrast to the white jersey, which also includes subtle imprints of Inter Miami's distinctflamingo logoacross the overallbody.

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Cystic fibrosis advocate and Australian of the Year award-winner Emmah Money on defying expectations – ABC News

Posted: at 11:03 pm

Updated February 28, 2020 11:32:05

"This attitude of having life expectancy never sat right with me," Emmah Money says. "And I never saw that as a reason not to have a baby."

Emmah has cystic fibrosis (CF), a hereditary illness that leaves people with an average 37-year life expectancy.

Born in 1987, her biological parents were told she would not even survive childhood and subsequently put her up for adoption.

But the infant had a glint in her eye suggesting she wanted to stick around and, at the age of 32 with a list of achievements behind her, was named South Australia's Local Hero during the 2020 Australian of the Year Awards in January.

It followed a hard-fought role in getting the critical CF drug, Orkambi, listed on Australia's Pharmaceutical Benefits Scheme, and launching CF Mummy, a social media brand supporting those with CF who want to have babies.

"I started CF Mummy when I fell pregnant with my daughter seven years ago," Emmah said.

"Wanting to have kids was almost unheard of because the attitude was: 'Why would you have a baby when you know you're going to be dead, or you're only going to make it to 30 if you're lucky?'.

"There was nothing that was positively supportive."

Emmah also talks in schools about overcoming obstacles such as bullying and depression subjects that resonate with her after repeatedly having her education and social development interrupted by illness and infection.

"One year I spent 16 weeks hospitalised and I was in primary school where you can imagine kids were finding their friends," she said.

"So having to be absent and then trying to come back and sit in on a social level, I did struggle."

One thing that makes CF difficult to understand for many people is it is an invisible disorder that damages the lungs, digestive system, and some internal organs, meaning its sufferers often appear no different to anybody else.

In high school, Emmah would have her school work sent to her during repeated hospital stays, meaning she could appear like she was getting special treatment for an "invisible illness".

"I fell very unwell when I got into high school and I was bullied very badly throughout," Emmah said.

"Everyone started getting their driver's licence and I would have girls follow me home and taunt me; they threw eggs on our loungeroom window.

"When you sort of stand out for whatever reason, it makes you a target."

At 16, Emmah was recognised for her work as a national youth ambassador for CF by Girlfriend Magazine, which awarded her Girlfriend of the Year.

It happened the same year that two friends she had grown up with died as a result of CF.

"So here's people at school giving me grief, and I remember thinking, 'God, if only you knew what I've had to go through'," Emmah said.

Her troubles were amplified when the Make a Wish Foundation set up a chance for Emmah to knock about with professional surfers Layne Beachley and Kelly Slater in Year 11, an adventure that resulted in Emmah being contracted to do some modelling for a surfwear label.

"I'd started a new school and was modelling surfwear clothing my photo was on a billboard and girls didn't like that."

"The sad thing is, being bullied in general will always be a part of your personal development, but I am who I am because I was bullied, to an extent."

Thanks to the recognition she received, Emmah had an autobiographical book, entitled The Words Inside, published when she was 17.

Another significant occasion that took place just before her 17th birthday was meeting her biological parents by chance, two people who believed she had passed away.

Emmah described them as "lovely people" but did not believe she would be here today if they had kept her.

"They were told, 'This baby will die', and they were not in a position to mentally be strong for me and give me the life I needed," Emmah said.

"They wanted to give me to a family that could actually give me the life I deserved."

She said the parents who brought her up were "wonderful and taught me many things".

"My mum told me they wanted a baby with special needs, so they went through a special needs adoption course," she said.

"Dad's a paraplegic but he represented Australia in the Seoul Paralympics in weightlifting in 1988.

"He remembers when they finally got me, he would stay up at night watching me sleeping in the bassinet because he was just in disbelief.

"They're amazing, and I look at the foundations they've given me: Dad's a paraplegic and he could have been depressed about it, but he's made the most of this life."

Emmah has a new-found confidence in managing her illness thanks to the Australian Government's 2018 decision to list the drug, Orkambi, on the Pharmaceutical Benefit Scheme after repeatedly rejecting it.

Until that moment, it cost up to $250,000 a year to access the drug, leaving it out of reach for most CF sufferers, including Emmah.

Australians can now access it for a maximum of $40.30 per script although there has been some controversy around access and who is entitled to it.

"There was probably three years of petitions, multiple applications and forms we'd submit to be reviewed by the Government, and I was also involved with quite a few protests," Emmah said.

"There was one in particular in Adelaide with some of the CF community members, and we walked through the streets just to get our voices heard.

"Because we got recognised on the news, it was louder than the petition itself."

Emmah started taking Orkambi within weeks of it being available in mid 2018, and while she endured the initial "rollercoaster" of side effects, such as tightness in her chest and migraines, she persisted and attributes the drug for getting her through a flu infection last year.

"The flu, for CF people, can kill you," Emmah explained.

"Because of Orkambi, that flu did not deteriorate my lungs or my health, and it allowed me to be stable."

A friend who was in hospital at the same time as her, however, was not so lucky.

Emmah spoke at his funeral in July.

"There's still a lot of suffering that goes on, and while Orkambi was a great win for quite a lot of us, it's not a cure," she said.

As an ambassador for Cure4CF, Emmah is organising a fundraiser for the CF national awareness month on May 29, or 65 Roses Day.

Her background and a "gift for the gab" has led Emmah to remain an ongoing advocate for CF sufferers, as well as being an "empowerment speaker".

She is working on a second autobiography about going through pregnancy and motherhood with CF and hopes to release a children's book later this year.

The defiance that pulled her through childhood is as strong as ever and she admits she never does anything by halves.

"When I was a child, I never had anybody with CF I could live up to because there wasn't anybody who lived long enough," Emmah said.

"The life expectancy has changed but for a very long time the number was 37, but I'm now 32 years old and I'm still going with my original lungs and have so much to give to this world."

Topics:government-and-politics,community-and-society,health,health-administration,health-policy,adelaide-5000,sa

First posted February 28, 2020 08:45:40

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Woman raises awareness of cystic fibrosis (CF) charity in memory of her cousin – Inverness Courier

Posted: February 15, 2020 at 11:01 pm

Emma Wilson with her cousin Jade Munro, who passed away in April 2019 after a lifelong struggle with cystic fibrosis.

An Inverness woman started fundraising for the Leanne Fund after her cousin lost her lifelong struggle with cystic fibrosis.

Emma Wilson (30) decided to run the Loch Ness Marathon last October, raising more than 2300 for the charity, which helps children suffering from CF and their families in the Highlands and Islands.

Jade Munro died aged 23 last April.

Ms Wilson said the cause was close to her heart: Jade was an amazing person and never complained about her condition even though she had to live with it her whole life.

She was so generous that before she died she asked to donate her beautiful hair to the Princess Trust to help kids with cancer.

She said it was the only thing she could donate as she couldnt become an organ donor because of her condition.

Ms Wilson knew about the Leanne Fund because Jade had received some support from it.

She said: The Leanne Fund does such a great job in helping children with CF, and its a local charity, so I felt I wanted to do something to help them.

I am a keen runner but I had never run a marathon before and it was more demanding than I had thought. Id like to do more fundraising activities in the future.

Ms Wilson has been helped by pupils at Cradlehall Nursery, who contributed to the fundraising effort by collecting almost 400 at their Christmas shows.

Chrisetta Mitchell, development manager at the Leanne Fund, said: We are so grateful to Emma for her support of the Leanne Fund in memory of her cousin Jade.

The incredible total raised from her marathon run will be used to provide vital services to those affected by cystic fibrosis.

The Leanne Fund is in its 10th anniversary year.

To find out more about the charity, visit http://www.theleannefund.co.uk and visit http://www.justgiving.com/fundraising/emma-wilson146 for Ms Wilsons Just Giving page where donations can be made.

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Ligand buys its way into partnership with Roche, CF Foundation plus some ion channel tech once owned by Pfizer – Endpoints News

Posted: at 11:01 pm

Ligand Pharma is once again buying its way into collaborations with some high-profile partners, feeding eight more drug discovery programs into its pipeline at a bargain price.

Its latest acquisition target is the core assets of Icagen, a neuroscience and rare disease-focused player headquartered in Durham, North Carolina. For $15 million upfront, Ligand is bagging one neurological program partnered with Roche, a cystic fibrosis project backed by the CF Foundation, as well as six wholly-owned assets.

Additional milestones and royalty earnouts will cap at $25 million.

Icagen has built deep expertise focused on ion channels and transporters to support target identification and evaluation, and has an established track record in ion channel drug discovery from screening to lead optimization, Ligand CEO John Higgins said in a statement. We expect these capabilities will be synergistic across multiple technology platforms at Ligand, particularly with Vernalis and in novel OmniAb antibody discovery targeting ion channels and transporters.

Vernalis was beat up by back-to-back FDA rejections when Ligand came to its rescue in August 2018, paying $43 million to get their hands on eight partnered programs and an operation in the UK.

Inking deals with companies skilled in later-stage drug development with the promise of royalties are core to Ligands business strategy one that short sellers have criticized as risky if they find the wrong partners.

Investors didnt appear impressed by Ligands choice, sending shares $LGND down 1.64% to $94.76.

Icagen, afterall, took a long and convoluted journey to this point. Having started out in 1992 and gone public in 2005, it sold itself to Pfizer in 2011 only to be reacquired and relaunched in 2015.

San Diego-based Ligand said it expects the deal to close in April, at which point it can push ahead with the preclinical programs at full speed.

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Proteostasis Therapeutics Announces Keystone Symposia Presentation of Organoid Study from More than 300 Adult CF Patients – Yahoo Finance

Posted: January 27, 2020 at 12:13 am

Ex vivo Data with Proteostasis' CFTR Modulators in non-F508del and non-G551D CF Genotypes Marks First Step Toward a Personalized Medicine Approach

BOSTON, Jan. 22, 2020 /PRNewswire/ --Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, today announced the presentation of results from an ex vivo study of the Company's proprietary cystic fibrosis transmembrane conductance regulator (CFTR) modulators in organoids from individuals with CF who are ineligible for the current standard of care CFTR modulator therapies due to their genotype, a population of approximately 2,300 adults in Europe alone. The results, outlined in a poster entitled "Intestinal Organoid Models as a Path for Personalized Therapy Development in Cystic Fibrosis," will be presented at the Keystone Symposia on Tissue Organoids taking place on January 19-23, 2020 in Vancouver, BC, Canada.

The study remains on track for collecting tissue samples from up to 500 CF patients with less common genotypes by the end of Q1 2020 for assaying as organoids and for testing responsiveness to investigational CFTR modulators, including Proteostasis' CFTR potentiator, corrector and amplifier, dirocaftor (DIR), posenacaftor (POS) and nesolicaftor (NES), respectively. Data from the organoid study will be used to select a subset of patients for a confirmatory clinical trial, known as the CHOICES trial (Crossover trial based on Human Organoid Individual response in CF - Efficacy Study). This organoid program is a strategic initiative funded by the European Commission, which has invited a select number of drug developers and leading researchers in CF to build a roadmap for personalized therapeutics in CF. Based on the outcome of the study, this transition from precision to personalized medicine for the treatment of CF could begin in patients with less common mutations.

The organoid study seeks to measure the ex vivo responsiveness to the PTI CFTR modulators in tissue samples collected via a rectal suction procedure. The rectal tissue is developed into an organoid or a miniaturized organ that is genetically identical to the patient donor and shares the same micro-anatomy as the organ from which they were derived. Organoid cultures frommore than370 adult CF patients have been established to date. Based on initial genotype analysis, approximately 85% of enrolled patients carry genotypes that lead to CFTR protein synthesis making them eligible for ex vivo study with DIR, POS and NES. Data from the organoid study will be used to select a subset of patients for the confirmatory CHOICES clinical trial. The poster outlining these results is available on the Company's website at proteostasis.com.

"With advancements in models such asorganoid testing used to predict the effectiveness of CFTR modulator treatments, the transition from precision to personalized medicine in this disease is an inevitability," saidGeoffrey Gilmartin, M.D., M.M.Sc., Chief Medical Officer ofProteostasis Therapeutics. "Essential to this transition is the introduction of more therapeutic options that expand the treatment choices for patients and physicians. As the only company in the HIT-CF consortium with a proprietary combination of novel CFTR modulators that have demonstrated positive Phase 2 data, we remain very enthusiastic about its progress and the translation of these results to the clinic in the CHOICES trial."

"Access to CFTR modulators in Europe is challenged by either the ineligibility of patients for approved drugs due to their genotype, or by the lack of reimbursement for approved drugs due to their high cost," saidCornelis K. van der Ent, Professor at the Department of Pediatric Pulmonology of the Wilhelmina Children's Hospital at the University Medical Center in Utrecht, the Netherlands and HIT CF Project Coordinator. "An organoid-assay-based personalized medicine approach offers the potential of a new decision-supporting technology to inform clinical decisionsand provide each patient with CFTR modulators that lead to their highest possible benefit."

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CHOICES, which is expected to initiate in mid-2020, will be the first ever personalized medicine-based study in CF. Fully funded by the HIT-CF, this trial is a placebo controlled, double blind, crossover study with an 8-week treatment period and 6 months of uninterrupted dosing. The CHOICES trial will complement the MORE trial (Modulator Options to RestorE CFTR study), a global, Phase 3, randomized, placebo-controlled study in CF subjects with the common F508del homozygous mutation, which is designed to confirm the positive efficacy and tolerability results from a recently completed Phase 2 study of the Proteostasis CFTR modulator triple combination.

About HIT-CF Europe

HIT-CF Europe is a research project which aims to provide better treatment and better lives for people with cystic fibrosis (CF) and rare mutations. To achieve this, drug candidates are first tested on patient-derived organoids in qualified laboratories across Europe. Subsequently, based on the measured signal in the organoids, a smaller group of patients will be invited to participate in a clinical trial. All participating centers are part of the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN). The project has received funding from the European Union's Horizon 2020 research and innovation program under grant agreement number 755021. For more information, visit http://www.hitcf.org.

About Proteostasis Therapeutics, Inc.

Proteostasis Therapeutics, Inc. is a clinical stage biopharmaceutical company developing small molecule therapeutics to treat cystic fibrosis and other diseases caused by dysfunctional protein processing. Headquartered in Boston, MA, the Proteostasis Therapeutics team focuses on identifying therapies that restore protein function. For more information, visit http://www.proteostasis.com.

Safe Harbor

To the extent that statements in this release are not historical facts, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as "aim," "may," "will," "expect," "anticipate," "estimate," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Examples of forward-looking statements made in this release include, without limitation, statements regarding the potential of our proprietary combination therapies for the treatment of CF, the potential benefit to patients of our proprietary combination therapies, expected timing of patient enrollment in our clinical studies and cohorts for our clinical programs, including our planned Phase 3 programs and initiation of registrational or pivotal studies. Forward-looking statements made in this release involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved. Such risks and uncertainties include, without limitation, the possibility final or future results from our drug candidate trials (including, without limitation, longer duration studies) do not achieve positive results or are materially and negatively different from or not indicative of the preliminary results reported by the Company (noting that these results are based on a small number of patients and small data set), uncertainties inherent in the execution and completion of clinical trials (including, without limitation, the possibility that FDA or other regulatory agency comments delay, change or do not permit trial commencement, or intended label, or the FDA or other regulatory agency requires us to run cohorts sequentially or conduct additional cohorts or pre-clinical or clinical studies), in the enrollment of CF patients in our clinical trials in a competitive clinical environment, in the timing of availability of trial data, in the results of the clinical trials, in possible adverse events from our trials, in the actions of regulatory agencies, in the endorsement, if any, by therapeutic development arms of CF patient advocacy groups (and the maintenance thereof), and those set forth in our Annual Report on Form 10-K for the year ended December 31, 2018, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2019 and our other SEC filings. We assume no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

CONTACTS:

Investors:David Pitts / Claudia StyslingerArgot Partners212.600.1902david@argotpartners.com/ claudia@argotpartners.com

Media:David RosenArgot Partners212.600.1902david.rosen@argotpartners.com

View original content:http://www.prnewswire.com/news-releases/proteostasis-therapeutics-announces-keystone-symposia-presentation-of-organoid-study-from-more-than-300-adult-cf-patients-300990860.html

SOURCE Proteostasis Therapeutics, Inc.

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Proteostasis Therapeutics Announces Keystone Symposia Presentation of Organoid Study from More than 300 Adult CF Patients - Yahoo Finance

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Point Leamington mother hopes Canada will green light latest cystic fibrosis treatment – The Telegram

Posted: at 12:13 am

NICHOLAS MERCER

THE CENTRAL VOICE

POINT LEAMINGTON, N.L. Kelly Earle is an expert in navigating the halls of the Janeway Childrens Hospital in St. Johns.

For the last year, Earle and her 13-year-old daughter Sammi-Jo James have visited the Janeway once a month for checkups. Those visits can turn into 14-day stays as James is treated for cystic fibrosis, a disease which attacks her lungs and digestive system.

The Point Leamington resident has no problem showing someone how to get to the cafeteria or find a certain hospital wing. When she escorts people around, she will tell herself she is a tour guide.

Earles probably seen too much of the Janeways hallways and waiting rooms.

While those trips to the Janeway are sure to continue well into 2020, Earle has some renewed hope that a new treatment for her daughter could be on the horizon.

A new drug named Trikafta was approved in the United States in October and it offers a game-changing treatment for those with cystic fibrosis. It has not been approved by Health Canada.

Under the Food and Drug Administration, Trikafta was approved for use by cystic fibrosis patients 12-years-old or older and who have the most common form of the disease.

James fits both of the criteria.

(Trikafta) is the only hope for Sammi other than therapy and a lung transplant, said Earle.

Earle joins a long list of Canadian patients who are calling for all levels of the Canadian government to bring this drug to the country.

An online petition started by Cystic Fibrosis Canada currently has over 31,000 pledges for action.

Earle has spoken to Coast of Bays-Central-Notre Dame MP Scott Simms, her federal representative, about helping to push the approval of the drug through Health Canada.

Sometimes Ill look at stuff about Trikafta on the computer and Ill have to walk away, said Earle. Ill think (Sammis health) could be changed in a couple of weeks.

Her daughter often stays in the same fourth floor room for her two week stays. They call it James bedroom.

Earle doesnt know the exact room number, but the way to it is singed into her memory.

She gets there by instinct and muscle memory.

If (James) gets admitted it is a relief, said Earle who always packs for a 14-day stay ahead of their monthly Janeway trip. I know she is going to get better.

Crashing down

James was diagnosed with cystic fibrosis at 33 months old. Until then, she always had health problems and couldnt keep food down.

The news sent shockwaves through Earles world.

My world crashed then, she said. Any normal that I knew before that was shattered.

Her life became filled with hospital trips as the Janeway, and Ronald McDonald House became a second home. It also filled with treatments at home and school.

James often experiences bouts of coughing that lead to nausea. She might lose a couple of pounds when this happens.

In November, she had a feeding tube placed in her stomach to deliver nutrients she needs. Along with medication, she uses this tube to feed for 10 hours at night.

CF is progressive, said Earle. The healthiest she is, is right now.

At the end of each day, Im thankful that we got through that day. Only thing I can say is that this is a good day.

Sometimes, James sheds tears as she goes through a procedure during those long hospital stays. They never last long and shell apologize shortly after.

She is a strong person and her mother marvels at her strength.

The things that Sammi have had to go through, me personally, I wouldnt be able to do them, said Earle.

Earle will catch herself watching her daughter as she scrolls through YouTube and becomes engrossed in her favourite videos.

Her mother will experience an explosion of memories. Shell see James cutting a cake as the 2019 Janeway Childrens Miracle Network champion and the subsequent trip to Florida they took because of it.

In her minds eye, Earle sees a smart and bubbly girl who meets every day with a smile.

You make it fun. You take lots of pictures, you make lots of memories and you have lots of fun, she said.

nicholas.mercer@thecentralvoice.ca

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Point Leamington mother hopes Canada will green light latest cystic fibrosis treatment - The Telegram

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