Category Archives: Cf

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

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

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

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

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

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

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

Originally posted here:

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

ValuEngine upgraded shares of CF Industries (NYSE:CF) from a hold rating to a buy rating in a research note issued to investors on Wednesday, January 8th, ValuEngine reports.

A number of other research firms have also recently issued reports on CF. Cowen upped their price target on shares of CF Industries from $59.00 to $62.00 and gave the company an outperform rating in a report on Thursday, September 26th. Zacks Investment Research cut shares of CF Industries from a buy rating to a hold rating and set a $49.00 target price for the company. in a research note on Monday, November 4th. Consumer Edge started coverage on shares of CF Industries in a research note on Monday, September 23rd. They issued an overweight rating for the company. Scotiabank cut shares of CF Industries from an outperform rating to a sector perform rating and set a $48.00 target price for the company. in a research note on Wednesday, September 18th. Finally, Bank of America raised shares of CF Industries from a neutral rating to a buy rating and set a $56.00 price target for the company in a research report on Monday, November 25th. One investment analyst has rated the stock with a sell rating, ten have assigned a hold rating and six have assigned a buy rating to the companys stock. The stock currently has an average rating of Hold and a consensus target price of $50.80.

Shares of NYSE CF traded down $1.57 during mid-day trading on Wednesday, reaching $40.93. The company had a trading volume of 2,503,762 shares, compared to its average volume of 2,277,601. The stock has a market capitalization of $9.29 billion, a PE ratio of 18.78, a price-to-earnings-growth ratio of 2.91 and a beta of 0.95. CF Industries has a twelve month low of $38.90 and a twelve month high of $55.15. The stocks 50 day moving average is $45.99 and its 200-day moving average is $47.41. The company has a debt-to-equity ratio of 0.74, a quick ratio of 1.10 and a current ratio of 1.34.

In related news, Director Stephen A. Furbacher sold 3,500 shares of the firms stock in a transaction on Tuesday, November 5th. The shares were sold at an average price of $47.50, for a total transaction of $166,250.00. Following the sale, the director now directly owns 49,187 shares in the company, valued at $2,336,382.50. The sale was disclosed in a document filed with the SEC, which is available at this hyperlink. Also, VP Bert A. Frost sold 13,000 shares of CF Industries stock in a transaction on Thursday, November 21st. The shares were sold at an average price of $45.50, for a total transaction of $591,500.00. Following the completion of the sale, the vice president now owns 95,072 shares in the company, valued at $4,325,776. The disclosure for this sale can be found here. 2.00% of the stock is currently owned by company insiders.

Several institutional investors have recently added to or reduced their stakes in the company. North Star Investment Management Corp. bought a new stake in shares of CF Industries during the 3rd quarter worth about $30,000. Huntington National Bank bought a new stake in shares of CF Industries during the 4th quarter worth about $31,000. Parallel Advisors LLC boosted its holdings in shares of CF Industries by 103.7% during the 3rd quarter. Parallel Advisors LLC now owns 725 shares of the basic materials companys stock worth $36,000 after buying an additional 369 shares during the period. CSat Investment Advisory L.P. boosted its holdings in CF Industries by 27.5% in the 2nd quarter. CSat Investment Advisory L.P. now owns 1,010 shares of the basic materials companys stock valued at $47,000 after purchasing an additional 218 shares during the period. Finally, B & T Capital Management DBA Alpha Capital Management bought a new stake in CF Industries in the 3rd quarter valued at about $60,000. Institutional investors own 93.48% of the companys stock.

About CF Industries

CF Industries Holdings, Inc manufactures and distributes nitrogen fertilizers and other nitrogen products worldwide. Its principal nitrogen fertilizer products include ammonia, granular urea, urea ammonium nitrate solution, and ammonium nitrate. The company also offers diesel exhaust fluid, urea liquor, nitric acid, and aqua ammonia products, as well as compound fertilizer products with nitrogen, phosphorus, and potassium.

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CF Industries (NYSE:CF) Rating Increased to Buy at ValuEngine - Slater Sentinel

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."

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 - BioSpace

CF Industries Holdings, Inc. [NYSE: CF] stock went down by -3.48% or -1.54 points down from its previous closing price of $44.25. The stock reached $42.71 during the last trading session. During the course of the last 5 trading session in the week, CF share price went up during 2 out of 5 days, that way losing -5.97% in the period of the last 7 days.

CF had two major price movements with the latest trading session as the price jumped to a high of $44.32, at one point touching $41.92. The latest high is set lower that the high price recorded in the period of the last 52 weeks, set at $44.32. The 52-week high currently stands at $55.15 distance from the present share price, noting recovery 2.03% after the recent low of $38.90.

Its stock price has been found in the range of 38.90 to 55.15. This is compared to its latest closing price of $44.25.

Keep looking out for the next-scheduled quarterly financial results that this company is expected to put out, which is slated to occur on Wed 12 Feb (In 21 Days).

Now lets turn to look at profitability: with a current Operating Margin for CF Industries Holdings, Inc. [CF] sitting at +15.67 and its Gross Margin at +20.46, this companys Net Margin is now 10.40%. These metrics indicate that this company is not generating as much profit, after accounting for expenses, compared to its market peers.

This companys Return on Total Capital is 6.37, and its Return on Invested Capital has reached 8.70%. Its Return on Equity is 8.87, and its Return on Assets is 2.16. These metrics show a mixed bag, which means that this investments attractiveness can be quickly increased or decreased in the short term, depending on future updates CF financial performance.

Turning to investigate this organizations capital structure, CF Industries Holdings, Inc. [CF] has generated a Total

What about valuation? This companys Enterprise Value to EBITDA is 8.96 and its Total Debt to EBITDA Value is 3.00. The Enterprise Value to Sales for this firm is now 3.00, and its Total Debt to Enterprise Value stands at 0.28. CF Industries Holdings, Inc. [CF] has a Price to Book Ratio of 3.28, a Price to Cash Flow Ratio of 6.75 and P/E Ratio of 19.58. These metrics all suggest that CF Industries Holdings, Inc. is more likely to generate a positive ROI.

Shifting the focus to workforce efficiency, CF Industries Holdings, Inc. [CF] earns $1,476,333 for each employee under its payroll. Similarly, this companys Receivables Turnover is 15.71 and its Total Asset Turnover is 0.33. This publicly-traded organizations liquidity data is also interesting: its Quick Ratio is 1.37 and its Current Ratio is 1.81. This company, considering these metrics, has a healthy ratio between its short-term liquid assets and its short-term liabilities, making it a less risky investment.

CF Industries Holdings, Inc. [CF] has 216.30M shares outstanding, amounting to a total market cap of $9.24B. Its stock price has been found in the range of 38.90 to 55.15. At its current price, it has moved down by -22.56% from its 52-week high, and it has moved up 9.79% from its 52-week low.

This stocks Beta value is currently 1.04, which indicates that it is 2.98% more volatile that the wider market. This stocks Relative Strength Index (RSI) is at 31.90. This RSI score is good, suggesting this stock is neither overbought or oversold.

Shares of CF Industries Holdings, Inc. [CF], on the whole, present investors with both positive and negative signals. Wall Street analysts have mixed reviews when it comes to the 12-month price outlook, and this companys financials show a combination of strengths and weaknesses. Based on the price performance, this investment is somewhat risky while presenting reasonable potential for ROI.

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CF Industries Holdings, Inc. [CF] Analyzing the CF using Fundamentals and Trend Analysis - The Dwinnex