Business Wire

Rare Diseases: Expected Benefits of a Physiological Absorption of Amino Acids in PKU Management. PKU GOLIKE® by APR Can Play a Role

Jaa

The recent article “Optimising amino acid absorption: essential to improve nitrogen balance and metabolic control in phenylketonuria”, published on the last issue of “Nutrition Research Reviews”, sheds the light on PKU unmet needs, opening up new interesting avenues in PKU dietary management.

The paper, by four renowned experts in the field of inherited metabolic diseases (Anita MacDonald, Rani H. Singh, Júlio C. Rocha and Francjan J. van Spronsen), highlights for the first time the expected important benefits for PKU patients of an amino acids (AA) formulation providing a physiological absorption profile of AA, similar to that of intact natural proteins (1).

The authors start from the consequences of the absorption profile of free AAs: as they are ready for absorption and bypass the digestive phase, free AAs intake is associated to a less efficient AA utilization, compared with intact protein sources, as well as excessive blood AA fluctuations.

Experts agree that protein substitutes with the ability to prolong absorption of AAs, mimicking physiological absorption kinetics of intact natural proteins, may allow a more efficient AA utilization, and thus contribute to support effectively normal growth and an overall healthy body composition.

Such prolonged-release formulations could play an important role in ameliorating the dietary management of PKU patients and provide them significant benefits. In particular, they could ideally simplify treatment by reducing the number of AA mixture administrations, and thus relieve the PKU burden. According to the conclusions of the article, positive consequences are also plausible on neurocognitive performance via a better balance in brain AA concentrations.

APR Applied Pharma Research s.a. (“APR”) is confident that PKU GOLIKE®, the innovative Phenylalanine (Phe)-free medical food formula for Phenylketonuria (PKU), could offer a new dietary treatment option to the benefit of patients and HCPs.

Leveraging on its extensive pharma development expertise, APR applied for the first time a pharmaceutical technology to a medical food - the patented Physiomimic Technology™, which can effectively prolong the release of the AAs and support their physiological absorption, mimicking that of intact proteins.

As showed in pre-clinical data generated independently by APR, the kinetic profile of the AAs engineered with the Physiomimic™ Technology resembles that of casein - a reference food protein known to have a prolonged absorption profile. Recent clinical data in human subjects confirmed the same kinetic absorption pattern of AAs with PKU GOLIKE® in favor of an improved efficiency in the use of AAs.

Further pre-clinical and clinical evidences will be generated by APR in the coming future to support the benefits of AAs physiological absorption associated with PKU GOLIKE® and the patented pharmaceutical Physiomimic Technology ™ behind it. Besides PKU GOLIKE®, other formulations are currently in the pipeline to convey similar benefits to other rare inherited metabolic disorders by applying the same pharmaceutical technology platform.

(1) Optimising amino acid absorption: essential to improve nitrogen balance and metabolic control in phenylketonuria, Anita MacDonald, Rani H. Singh, Júlio César Rocha and Francjan J. van Spronsen - Nutrition Research Reviews 10/2018

About Phenylketonuria or PKU

Phenylketonuria or PKU is a rare, genetic, recessive metabolic disorder affecting about 50.000 people worldwide. PKU is characterized by the deficiency or the malfunctioning of a liver enzyme needed to process phenylalanine (“Phe”), an essential amino acid found in most protein-containing food. Excessive amounts of Phe in the bloodstream become toxic to the brain, impairing the normal development of the central nervous system. PKU can only be treated through a strict, life-long, low-protein (low-Phe) dietetic treatment combined with a daily assumption of low-protein modified foods and medical food, which provides Phe-free amino acids and other important nutrients, needed due to the dietary restrictions of PKU patients.

About PKU GOLIKE®

PKU GOLIKE is an innovative food for special medical purposes (FSMP) consisting of a Phe-free amino acid mixture. For the first time a pharmaceutical technology has been applied to a FSMP and the innovative PKU GOLIKE, engineered with Physiomimic technology, provides a prolonged-released of amino acids and grants a remarkable taste and odor masking while preventing aftertaste. This prolonged release leads to a physiological absorption of amino acids, similar to dietary proteins, to favor a more efficient amino acid utilization and health benefits. On the other hand, the improvement of free-amino acids' organoleptic features aims to support compliance to the nutritional management of the diet for a better quality of life.

The product line is formed by: PKU GOLIKE PLUS 3-16 & PKU GOLIKE PLUS 16+ with amino acids, vitamins & minerals, and PKU GOLIKE PURE 3+ with only amino acids.

For more information about PKU GOLIKE® and the Physiomimic™ Technology, please visit: www.apr.ch/apr-pharma-products/medical-prescription/genetic-metabolic-disease/

About APR Applied Pharma Research s.a.

APR is a Swiss independent pharma company focused on development and commercialization of innovative, science-driven products designed to address unmet needs in niche or rare therapeutic areas on a global basis. APR combines drug development expertise with proprietary drug delivery technologies to deliver to patients suffering from rare diseases with solutions meaningfully improving their life and empowering caregivers with better ways to manage such rare diseases. APR has a balanced portfolio of revenue generating products marketed in all major markets, combined with a compelling pipeline of innovative products at different stages of development, specifically in the treatment of rare recessive metabolic disorders, rare dermatological and ocular diseases. APR’s products are commercialized by APR directly through its own sale and marketing teams in selected countries of Europe as well as through a solid global network of commercial partners.

Contact information

APR Applied Pharma Research s.a.:
Paolo Galfetti, Chief Executive Officer
paolo.galfetti@apr.ch
+41 91 6957020

Tietoja julkaisijasta

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

Tilaa tiedotteet sähköpostiisi

Haluatko tietää asioista ensimmäisten joukossa? Kun tilaat mediatiedotteemme, saat ne sähköpostiisi välittömästi julkaisuhetkellä. Tilauksen voit halutessasi perua milloin tahansa.

Lue lisää julkaisijalta Business Wire

Warm welcome for ABB Formula E in Davos19.1.2019 12:00Tiedote

Just a few days before the start of the World Economic Forum 2019, Davos is celebrating a world premiere: The latest ABB FIA Formula E racing car - dubbed Gen2 – was transported on a train from the iconic Swiss Rhaetian Railway to Europe’s highest city, where it will be an exciting eyecatcher during the coming week at the Davos Platz railway station. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190119005003/en/ ABB FIA Formula E racing car in Davos (Photo: Business Wire) From this Saturday until next Sunday, January 27, the Gen2 will be on display in a special glass case. Interested passersby can learn about the ABB FIA Formula E racing series as well as about the activities of ABB in e-mobility via an information system. With over 8,500 fast charging stations installed in 70 countries around the world, ABB is the global market leader for charging infrastructures. To watch the video, click here ABB (ABBN: SIX Swiss Ex) is

PMI’s Mission Winnow Goes Full Throttle with Ducati Corse for 2019 MotoGP™18.1.2019 20:00Tiedote

Philip Morris International Inc. (PMI) (NYSE: PM) is pleased to announce that its Mission Winnow initiative is expanding in 2019 to include the Ducati Corse racing team. Mission Winnow is a PMI-led campaign to raise global awareness of our passion and determination to constantly improve and evolve, as well as highlight the power of science, technology and innovation to build a better future. PMI has partnered with Ducati Corse since 2002 and has extended the relationship for another three years until the end of 2021. As of the start of this year’s MotoGP season, the team will be officially known as Mission Winnow Ducati. Mission Winnow was first launched in October 2018 with Scuderia Ferrari Mission Winnow, which – like Ducati Corse – has a passion for innovation and a relentless drive to improve in the team’s pursuit of victory. Ducati Corse is one of the most inspiring and resilient teams in MotoGP, with a 70-year history in racing. Ducati fans form a passionate community who appreci

IFF’s Frutarom Division Completes Acquisition of 60% of Thailand-based Mighty18.1.2019 15:50Tiedote

Regulatory News: International Flavors & Fragrances Inc. (NYSE:IFF) (Euronext Paris:IFF) (TASE:IFF), announced that its Frutarom Division has completed the acquisition of 60% of the share capital of The Mighty CO. LTD. (“Mighty”), a leading savory solutions provider in Thailand, thus continuing its growth strategy in Southeast Asia. Amos Anatot, President of IFF's Frutarom Division, said, “The completion of this deal with Mighty underscores that the Frutarom division will continue on its growth strategy and pursue attractive companies that create new opportunities or build on current capabilities.” Mr. Anatot continued, “And in this case, we are helping to grow our capabilities in savory solutions – already an area of strength for legacy Frutarom, now IFF." Mighty, founded in 1989, develops, produces and markets reaction flavors, with particular expertise in savory solutions. The company’s portfolio includes flavors, seasoning blends, marinades, and specialty functional raw materials f

LTI Q3 FY19: Constant currency revenue growth up 6.1% QoQ and 20.6% YoY; Net Profit jumps 32.8% YoY18.1.2019 15:02Tiedote

Larsen & Toubro Infotech (BSE code: 540005, NSE: LTI), a global technology consulting and digital solutions company, announced its Q3 FY19 results today. In US Dollars: Revenue at USD 346.9 million; growth of 5.6% QoQ and 18.2% YoY Constant Currency Revenue growth of 6.1% QoQ and 20.6% YoY In Indian Rupees: Revenue at Rs 24,729 million; growth of 6.1% QoQ and 31.3% YoY Net Income at Rs 3,755 million; growth of (6.2%) QoQ and 32.8% YoY “We are pleased to deliver another strong quarter with 5.6% QoQ growth in USD revenues. Our broad-based revenue growth, superior margin delivery and steady cash generation in Q3 is a testimony of our focused execution and client centricity. We are also thrilled to welcome Ruletronics to LTI family. Ruletronics enables businesses to transform and evolve digitally by providing innovative BPM and CRM solutions leveraging Pega Platform.” - Sanjay Jalona, Chief Executive Officer & Managing Director, LTI Recent Deal Wins Nets, the leading payments company in th

Schlumberger Announces Full-Year and Fourth-Quarter 2018 Results18.1.2019 15:00Tiedote

Schlumberger Limited (NYSE: SLB) today reported results for full-year 2018 and the fourth quarter of 2018. (Stated in millions, except per share amounts) Full-Year Results Twelve Months Ended Change Dec. 31, 2018 Dec. 31, 2017 Year-on-year Revenue $32,815 $30,440 8% Pretax operating income $4,187 $3,921 7% Pretax operating margin 12.8% 12.9% -12 bps Net income (loss) - GAAP basis $2,138 $(1,505) n/m Net income, excluding charges & credits* $2,261 $2,085 8% Diluted EPS (loss per share) - GAAP basis $1.53 $(1.08) n/m Diluted EPS, excluding charges and credits* $1.62 $1.50 8% Full-Year Consolidated Revenue by Area North America $11,984 $9,487 26% Latin America 3,745 3,976 -6% Europe/CIS/Africa 7,158 7,072 1% Middle East & Asia 9,543 9,394 2% Other 385 511 n/m $32,815 $30,440 8% North America revenue $11,984 $9,487 26% International revenue $20,446 $20,442 - North America revenue, excluding Cameron $9,668 $7,518 29% International revenue, excluding Cameron $17,675 $17,423 1% *These are non

FLIR Systems Awarded $89 Million Contract from French Armed Forces to Deliver Black Hornet Personal Reconnaissance System18.1.2019 14:00Tiedote

FLIR Systems, Inc. (NASDAQ: FLIR) announced today it has been awarded a contract from the French Defense Procurement Agency (DGA) in support of the French Operational Pocket Drone (DrOP) program. The contract has a ceiling value of $89 million to provide the FLIR Black Hornet® 3 nano-unmanned aerial vehicle (UAV) and Personal Reconnaissance System (PRS) to support French Armed Forces operations. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190118005085/en/ The French Armed Forces awarded FLIR Systems a contract to deliver the Black Hornet Personal Reconnaissance System for military operations. (Photo: Business Wire) The Black Hornet PRS is the world’s smallest combat-proven nano-Unmanned Aerial System (UAS) and is currently deployed in more than 30 countries. The Black Hornet enables the warfighter to maintain situational awareness, threat detection, and surveillance no matter where the mission takes them. Equipped with el

Uutishuoneessa voit lukea tiedotteitamme ja muuta julkaisemaamme materiaalia. Löydät sieltä niin yhteyshenkilöidemme tiedot kuin vapaasti julkaistavissa olevia kuvia ja videoita. Uutishuoneessa voit nähdä myös sosiaalisen median sisältöjä. Kaikki STT Infossa julkaistu materiaali on vapaasti median käytettävissä.

Tutustu uutishuoneeseemme