PGES

NIJMEGEN, Netherlands, Nov. 1, 2023 /PRNewswire/ -- Khondrion, a clinical stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial diseases, today announced that the United States Patent and Trademark Office and the European Patent Office have granted patents covering the use of Khondrion's lead compound sonlicromanol as an [mPGES-1 inhibiting] anti-inflammatory agent. Sonlicromanol works in three distinct ways by modulating oxidative and reductive distress, along with anti-inflammatory properties. The compound is currently under investigation for its effectiveness in treating adult patients diagnosed with primary mitochondrial disease due to the m.3243A>G mutation in their mitochondrial DNA. This mutation is linked to various conditions such as classical MELAS and MIDD syndromes, CPEO, and mixed phenotypes. Sonlicromanol targets the key underlying mechanisms shared by many mitochondrial diseases, including high levels of reactive oxygen species, disrupted redox metabolism, and inflammation induced by PGE2. Research has demonstrated that sonlicromanol acts as a selective inhibitor of microsomal Prostaglandin E-synthase 1 (mPGES-1), contributing to its anti-inflammatory impact. Preclinical studies conducted in other disease models with elevated levels of cellular mPGES-1, such as particular prostate cancer cells, have displayed encouraging outcomes. These findings suggest a wider range of potential applications for sonlicromanol beyond its initial focus on primary mitochondrial diseases. "The distinctive triple mode of action of sonlicromanol has the potential to play an important role not just in primary mitochondrial diseases but also in in a range of rare and more prevalent disorders," said Prof. dr. Jan Smeitink, Chief Executive Officer at Khondrion. "This US and European patent issuance significantly strengthen our intellectual property portfolio and stands as a crucial step in the broader development of sonlicromanol." The US patent titled "Compounds as mPGES-1 inhibitors" was issued as U.S. patent No. 11,672,787 on June 13, 2023. Its European equivalent was granted on October 19, 2023 as EP 18808288. About Khondrion Khondrion is a clinical stage biopharmaceutical company developing therapies for patients with inherited mitochondrial diseases. Based on proprietary science and a deep biological understanding of mitochondrial dysfunction, the company is advancing its lead drug candidate sonlicromanol. Sonlicromanol is a first-in-class, oral small molecule targeting key underlying mechanisms of primary mitochondrial disease based on its uniquely differentiated triple mode of action: reductive distress modulation to help restore the cell's metabolism, oxidative distress modulation preventing ferroptotic cell death, and selective mPGES-1 inhibition resulting in anti-inflammatory effects. Based on the favourable benefit-risk profile shown across the clinical studies performed so far, Khondrion is finalizing the design of a global Phase 3 study which is currently foreseen to start in H2 2024. The compound has been granted orphan drug designations for the treatment of MELAS, Leigh disease and patients with maternally inherited diabetes and deafness (MIDD) in Europe, and for all inherited mitochondrial respiratory chain disorders in the US. It has also been granted a Rare Pediatric Disease designation in the US for the treatment of MELAS. Ongoing preclinical research of sonlicromanol shows potential broader applications of the drug in other rare diseases and more common disorders. For more information visit . About mitochondrial disease Mitochondrial disease is caused by defective mitochondria, which are present in all cells of the human body and are responsible for generating the energy necessary for cells to function. This can lead to a wide range of serious and debilitating illnesses that can appear shortly after birth or later in life. Signs and symptoms of these conditions may include issues with thinking, learning difficulties, blindness, deafness, heart failure, diabetes, fatigue, intolerance to exercise, muscle weakness walking difficulties, and stunted growth. Orphan diseases of the oxidative phosphorylation system like Leigh disease, MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders including MIDD (maternally inherited diabetes and deafness), and other respiratory chain / oxidative phosphorylation disorders, are all examples of mitochondrial disease. MELAS spectrum disorders are some of the most frequently observed primary mitochondrial diseases, in which all patients are characterized by an underlying point mutation (m.3243A>G) in the maternally inherited MT-TL1 gene. Forward-looking statements This press release may contain certain forward-looking statements regarding, among other things, the results, conduct, progress and timing of the company's clinical trials and presentation of data from clinical trials for sonlicromanol. Although the company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the company's control. These statements may include, without limitation, any statements preceded by, followed by or including words such as "target," "believe," "expect," "aim," "goal," "intend," "may," "anticipate," "foreseen," "estimate," "plan," "project," "will," "can have," "likely," "potential," "should," "would," "could" and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the company's control that could cause the company's actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. Except as required by law, the company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Contacts: Khondrion BV Prof. Dr. Jan Smeitink, CEO E-mail: [email protected] Tel: +31-24-7635000 LifeSpring Life Sciences Communication, Amsterdam Leon Melens E-Mail: [email protected] Tel: +31-6-53816427 Logo - SOURCE Khondrion

Khondrion announces sonlicromanol Phase IIb progress supporting Phase III development in MELAS spectrum disorders Sonlicromanol for MELAS spectrum disorders – a rare, progressive primary mitochondrial disease – to move into a Phase III registrational study supported by favourable benefit-risk profileBoth randomised placebo-controlled Phase IIb study and ongoing Phase IIb open label extension study show statistically significant and/or clinically meaningful results in multiple outcome measures with acceptable safety profileThough 28-day Phase IIb study did not meet primary endpoint of the attention domain score of cognitive functioning, the study demonstrated positive treatment effects in several other cognition and mood-related secondary endpointsPositive results on cognition, mood, quality of life, pain, fatigue and balance control were observed after up to 52 weeks sonlicromanol treatmentTopline data to be presented today by Khondrion’s CEO, Prof. Dr. Jan Smeitink, at Wellcome’s Mitochondrial Medicine - Therapeutic Development Conference, Cambridge, UK NIJMEGEN, the Netherlands – 22 November 2022: Khondrion, a clinical stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial diseases, today provides an update from (A) the 28-day double-blind, randomised, placebo-controlled, three-way cross-over KHENERGYZE Phase IIb study in 27 patients, and (B) the ongoing, open label extension KHENEREXT Phase IIb study (7 patients up to 52 weeks) (both studies jointly the “Phase IIb programme”). Sonlicromanol, Khondrion’s wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA. In line with previous studies, sonlicromanol was found to be safe and well tolerated in the Phase IIb programme, with no serious adverse effects. The overall evaluation of the shorter and longer term data from the programme shows patient benefits in several domains (cognition and mood, pain, fatigue and balance control) and marked improvements in global health and quality of life assessments, supporting sonlicromanol’s progression into Phase III clinical development in patients with MELAS spectrum disorders. In line with expectations in these slowly progressing rare diseases, analysis of the data shows more pronounced treatment effects after 52 weeks. These longer-term patient data are, therefore, instrumental in providing valuable insights for the design of the upcoming Phase III trial. Dr. Mirian Janssen, one of the principal investigators, Radboud Centre for Mitochondrial Medicine (Nijmegen, the Netherlands), said: ‘’I am encouraged by these promising results, especially the improvement in quality of life measurements and NMDAS score over a longer period. The fact that the study-patients wish to continue using sonlicromanol and their personal motivation are also positive signs. I have had the privilege to work closely with many of these patients for many years and I am pleased to observe tangible improvements in those patients who have now been treated with sonlicromanol for 52 weeks. I praise Khondrion for its pursuit of mitochondrial disease innovation, care to the patients in its trials and commitment to this development programme.” Phase IIb programme results A. KHENERGYZE Phase IIb study in m.3243A>G MELAS spectrum disorders (adults) Patients (n=27) treated with sonlicromanol (50 mg and 100 mg bid) for 28 days did not achieve a statistically significant improvement in the attention domain score of cognitive functioning, as assessed by the computerised Cogstate visual identification test, compared to those who received placebo.Post-hoc analyses of the Phase IIb study data showed positive trends in several other cognition and mood-related secondary endpoints as well as in other domains including fatigue.More specifically, statistically significant and clinically meaningful treatment effects versus placebo were obtained, amongst others for the Beck Depression Inventory (BDI, 100 mg bid, p=0.01) and the Cognitive Failure Questionnaire (CFQ, 100 mg bid, p=0.007).The totality of data suggests better effectiveness of the 100 mg bid dose relative to 50 mg bid.Acceptable safety profile confirmed. B. KHENEREXT Phase IIb open label extension study (ongoing) An interim analysis from the ongoing Phase IIb open label extension study, examining the long-term safety and a broad set of efficacy parameters of sonlicromanol (100 mg bid) in patients who completed the KHENERGYZE Phase IIb study, confirmed the positive results shown in the Phase IIb study on BDI and CFQ over a longer period of time in a first cohort of 7 patients who received sonlicromanol for 52 weeks.On the widely-used NMDAS score, specifically developed and validated to assess disease severity of adult patients with primary mitochondrial disease, the majority of subjects showed a meaningful improvement with a decrease up to 6 points after 52 weeks. In a Natural History Study conducted by De Laat et al. (J Med Genet 2021), it was observed that the NMDAS score in patients with m.3243A>G increased on average with 0.47 points per year, representing disease deterioration.Regarding other global health and quality of life related outcome measures, like RAND SF36 and EQ-5D-5L, the majority of patients overall who were affected for these parameters at baseline showed clinically meaningful responses, while both the clinician- and patient-reported global impression of change improved in the majority of patients in an otherwise progressive disease.In addition, the interim analysis showed marked improvements in multiple other outcome measures, including pain (Short-form McGill Pain Questionnaire), fatigue (Neuro-QoL short form) and balance control (Mini-Balance Evaluation Systems Test), with various endpoints showing statistically significant and/or clinically meaningful treatment effects.Acceptable safety profile confirmed. Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: “These results are an important milestone for Khondrion. While sonlicromanol’s effect on the primary endpoint within a 28-day period was insufficient in the Phase IIb trial, we believe the broader results from the Phase IIb programme, now up to 52 weeks, provide unequivocal evidence of the positive clinical impact and important patient benefit that sonlicromanol can provide. We are particularly encouraged by the positive signals on overall disease severity, mood, cognition, fatigue, balance and pain, all belonging to the most burdensome symptoms patients experience in their daily lives, as mentioned in UMDF’s Voice of the Patient Report 2019. We look forward to discussing our plans with European and U.S. regulators in the near future, ahead of the initiation of the Phase III registrational trial which is currently foreseen in late 2023. “I would like to express my heartfelt gratitude to each of the patients, and their families who have played a part in these studies, and to the patient advocacy groups, the clinical study teams in Nijmegen, Munich, Newcastle and Copenhagen, and Foundations and Governmental bodies for their special contributions to our research. And a huge thank you to the talented team at Khondrion for their continued hard work, furthering this field of research, and for their commitment to finding the new treatments that are so needed for patients.” A presentation of topline data from this sonlicromanol Phase IIb programme update will be given by Prof. Dr. Jan Smeitink today at Wellcome’s Mitochondrial Medicine – Therapeutic Development Conference, Cambridge, UK. The full data will be submitted for peer-reviewed publication. – ENDS – Notes to editors About the Phase IIb programme in adult patients with MELAS spectrum disorders KHENERGYZE – A 28-day double-blind, randomised, placebo-controlled, multi-centre, three-way cross-over Phase IIb study examining cognitive function in adult patients with a specific genetically confirmed DNA mutation in the mitochondrial transfer RNALeu(UUR) (MT-TL1m.3243A>G). This mutation is responsible for MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders, including classical MELAS, MIDD (maternally inherited diabetes and deafness) syndromes, and mixed phenotypes. Further details are available on clinicaltrials.gov (NCT04165239) and eudract.ema.europe.eu (2019-000599-40). KHENEREXT – An open-label extension study, enabling continued sonlicromanol treatment for patients who had completed the KHENERGYZE Phase IIb study and the collection of longer-term data. Alongside safety, the study also gathers a broad set of primary and secondary functional clinical outcome measures, including cognition, fatigue and motor-function related parameters. Further details are available on clinicaltrials.gov (NCT04604548) and eudract.ema.europe.eu (2020-000832-23). Additional pediatric study (ongoing) KHENERGYC – A placebo-controlled, double-blind Phase II study to explore the pharmacokinetics, safety and efficacy of sonlicromanol in children with a genetically confirmed primary mitochondrial disease and suffering from motor symptoms. The 6-month study, supported by Dutch Patient Foundations and an EFRO Grant (PROJ-00582), is investigating the effect of sonlicromanol in 24 children (from birth to 17 years) with genetically confirmed mitochondrial disease of which the gene defect is known to hamper the functioning of one or more oxidative phosphorylation system enzymes and who are suffering from motor symptoms. The study’s primary objective is to evaluate the effect of sonlicromanol on motor function using a range of validated, quantitative assessments including the Gross Motor Function Measure-88 and the Nine Hole Peg Test. Further details are available on clinicaltrials.gov (NCT04846036) and eudract.ema.europe.eu (2020-003124-16). About Khondrion Khondrion is a clinical stage biopharmaceutical company developing therapies for patients with inherited mitochondrial diseases. Based on proprietary science and a deep biological understanding of mitochondrial dysfunction, the company is advancing its lead drug candidate sonlicromanol. Sonlicromanol is a first-in-class, oral small molecule targeting key underlying mechanisms of mitochondrial disease based on its uniquely differentiated triple mode of action: reductive distress modulation to help restore the cell’s metabolism, oxidative distress modulation preventing ferroptotic cell death, and selective mPGES-1 inhibition resulting in anti-inflammatory effects. One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has recently completed a Phase IIb study in adult patients with m.3243A>G MELAS spectrum disorders. In combination with interim 52-week data from the ongoing Phase IIb open-laben extension study, analysis of the results has shown marked improvements in multiple endpoints which merit further study in a Phase III registrational trial. Sonlicromanol is also studied in a 6-month Phase II study in children with genetically confirmed primary mitochondrial diseases and who suffer from motor symptoms. The compound has been granted orphan drug designations for the treatment of MELAS, Leigh disease and patients with maternally inherited diabetes and deafness (MIDD) in Europe, and for all inherited mitochondrial respiratory chain disorders in the US. It has also been granted a Rare Pediatric Disease designation in the US for the treatment of MELAS. Sonlicromanol and other compounds from Khondrion’s proprietary library have the potential to be developed for a wide range of diseases and conditions with the aim of benefiting patients whose daily lives are severely impacted by mitochondrial impairment. For more information visit www.khondrion.com. About mitochondrial disease Mitochondrial disease occurs when mitochondria, found within all cells of the human body and responsible for producing the energy necessary for cells to function, are defective. This can result in a wide range of serious and debilitating illnesses occurring shortly after birth or later in life. Signs and symptoms of these can include cognitive problems, learning disabilities, blindness, deafness, heart failure, diabetes, fatigue, intolerance to exercise, muscle weakness and gait problems, and stunted growth. Orphan diseases of the oxidative phosphorylation system like Leigh disease, MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders including MIDD (maternally inherited diabetes and deafness), and other respiratory chain / oxidative phosphorylation disorders, are all examples of mitochondrial disease. MELAS spectrum disorders are some of the most frequently observed primary mitochondrial diseases, in which all patients are characterised by an underlying point mutation (m.3243A>G) in the maternally inherited MT-TL1 gene. Forward-looking statements This press release may contain certain forward-looking statements regarding, among other things, the results, conduct, progress and timing of the company’s clinical trials and presentation of data from clinical trials for sonlicromanol. Although the company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the company’s control. These statements may include, without limitation, any statements preceded by, followed by or including words such as “target,” “believe,” “expect,” “aim,” “goal,” “intend,” “may,” “anticipate,” “foreseen,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “potential,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the company’s control that could cause the company’s actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. Except as required by law, the company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Contacts: Khondrion BV Prof. Dr. Jan Smeitink, CEO E-mail: info@khondrion.com Tel: +31-24-7635000 www.khondrion.com Consilium Strategic CommunicationsDavid Daley, Melissa Gardiner, Kris LamE-mail: khondrion@consilium-comms.comTel: +44 20 3709 5700

Switzerland’s DBI announced a collaboration with life sciences giant Bayer AG, based in Germany. The companies will collaborate on the SPRAY SMART study of a new nasal drug for Obstructive Sleep Apnea Syndromes (OSAS). The goal of the collaboration between DBI and BAYER is to broaden insight into the dynamics of metabolites whose levels in the exhaled breadth of patients with OSAS are changed by the disease and in response to therapeutic intervention. Applying DBI’s technology and algorithm could provide early signs of treatment efficacy and potentially help the identification of sub-populations of OSAS patients responsive to specific treatments. DBI combines Secondary Electrospray Ionization (SESI) and High-Resolution Mass Spectrometry with advanced bioinformatics tools to provide unprecedented breath metabolic profiling. This allows for real-time breath analysis and study molecules that are untraceable by traditional methods. It opens a new path to advancing precision medicine and improving future clinical outcomes. Bayer’s first clinical study applying DBI’s technology is the placebo controlled SPRAY-SMART study. The apnea-hypopnea-index (AHI) will be measured to determine the severity of OSA after treatment with a new nasal spray for OSA treatment. The drug aims at preventing the collapse of the upper airways as one pathological mechanism in OSA. Elsewhere around the globe: LivaNova PLC – London-based LivaNova received the greenlight from the FDA to begin its investigational device exemption (IDE) clinical study, “Treating Obstructive Sleep Apnea using Targeted Hypoglossal Neurostimulation (OSPREY).” The OSPREY study will seek to demonstrate the safety and effectiveness of the aura6000 System, the LivaNova implantable hypoglossal neurostimulator intended to treat adult patients with moderate to severe obstructive sleep apnea (OSA). An innovative alternative to the traditional continuous positive airway pressure (CPAP) machine, the aura6000 System generates stimulation through a programmable, rechargeable and implantable pulse generator (IPG). The IPG is implanted in a subcutaneous pocket near the clavicle of the patient via an outpatient surgery. Mild stimulation pulses from the IPG are delivered via a lead to the hypoglossal nerve to stimulate the tongue during sleep and help keep the patient’s airway open. Dxcover Limited – Scotland’s ClinSpec Diagnostics has rebranded as Dxcover Limited. The company continues to focus on become a world-leader in liquid biopsy and artificial intelligence for early detection of cancer and other diseases. Dxcover is a spinout from the University of Strathclyde. Its technology has received the backing from well-known investors, including Scottish Enterprise, Mercia Asset Management, Norcliffe Capital, SIS Ventures, Eos Advisory and the University of Strathclyde. Coinciding with its rebranding, the company has taken over a 2,000-square-foot lab in the University’s Royal College Building in the heart of the Glasgow City Innovation District. At three times the size of Dxcover’s previous labs, the new site will accommodate the company’s ongoing growth, but will also permit its critical development work to continue while social distancing measures remain in place. Debiopharm– Switzerland-based Debiopharm announced Phase II results of a clinical study assessing naratuximab emtansine for the treatment of DLBCL and other B-cell malignancies. In the trial, naratuximab emtansine, which targets the CD37 antigen on the surface of B cells, was paired with rituximab, an anti-CD20 antibody. Data from the study revealed the combination treatment demonstrated meaningful efficacy and high complete response rates (CRR), especially in heavily pre-treated patients with more than two prior lines of treatment. The Objective Response Rate (ORR) in all efficacy-evaluable patients was 44.7%, complete response rate (CRR) was 31.6%. In patients with two or more prior therapies, non-primary refractory ORR was 46.4% and CRR was 32.1%. Veraxa Biotech GmbH – Formerly known as Velabs, Germany-based Veraxa forged a partnership with Swiss-based Quadira Biosciences Ag to develop a suite of novel ADCs for the treatment of various cancers. The collaboration will leverage both the advantages of Veraxa’s proprietary position-true click-chemistry-based drug conjugation technology in combination with Quadira’s superior 3D cellular assay and assessment systems. RhoVac AB – Sweden’s RhoVac will initiate a follow-up study of patients that took part in a Phase I/II prostate cancer trial assessing RV001. The objective of the long-term follow study, to be conducted in the coming months, is to find out to what extent these patients still have the appropriate level of RhoC specific immune response, what their PSA development has been, and to what extent they have progressed to additional therapy. Following a one-year review, the company’s drug candidate was considered safe and also induced a prevalent and long lasting immune RhoC-specific immune response in 86% of the patients who participated. The Phase IIb study is to show that RV001 can significantly prevent or procrastinate disease progression in these patients, something for which no standard therapy is available today. AlzeCure Pharma AB – A presentation at the IASP 2021 World Congress on Pain showed the company’s preclinical TrkA-NAM asset for osteoarthritic pain and other severe pain disorders is a promising new treatment. The company concluded that potent TrkA-NAM’s could potentially avoid some of the side effects observed for anti-NGF antibodies due to a more selective mechanism of action, while retaining the analgesic efficacy. Gesynta Pharma AB – Sweden-based Gesynta submitted an Investigational New Drug Application to the U.S. Food and Drug Administration for its oral drug candidate GS-248 in patients with systemic sclerosis. The study investigates the safety of GS-248 and its efficacy on Raynaud’s phenomenon and peripheral vascular blood flow. Top-line data is expected in the first quarter of 2022. GS-248 provides a combination of anti-inflammatory, vasodilatory and platelet inhibitory effects by potently and selectively inhibiting microsomal prostaglandin E synthase-1 (mPGES-1). The randomized, placebo-controlled, double-blind Phase II study will include approximately 80 patients at clinical sites in four European countries. Patients will receive GS-248 orally once daily, or placebo, for four weeks. Enterprise Therapeutics Ltd. – Based in the U.K., Enterprise Therapeutics dosed the first patients in a Phase I study for its novel inhaled cystic fibrosis therapy, ETD001. The first-in-man safety study is being conducted in healthy participants. ETD001 is an ENaC ion channel inhibitor with best-in-class potential aimed at treating all people with CF. ETD001 has previously been shown to have long duration of action in the lung and is therefore expected to provide a superior efficacy and safety profile compared to other ENaC drug candidates. ETD001 targets the epithelial sodium ion channel ENaC, which controls fluid volume and mucus clearance from the airways. By increasing the amount of airway fluid available to hydrate mucus, ETD001 addresses the underlying mechanisms of mucus congestion, and is expected to restore lung function, reduce the frequency of lung infections and improve patient quality of life. The Native Antigen Company – Also based in the U.K, the Native Antigen Company won ‘The Best COVID-19 Responder’ at OBN 2020 Awards. The award recognizes the company’s outstanding contribution to the fight against COVID-19. Domainex – The OBN awards also recognized U.K.-based Domainex as the best contract research organization. Over the past year, Domainex has made significant investment in new technology to support its clients’ research needs. The company grew its top line by approximately 45% in 2020 and welcomed over 30 new colleagues to its team to further extend its drug discovery offering, despite the challenges faced as a result of the pandemic. SAGA Diagnostics AB – Sweden’s SAGA announced that it has successfully been accredited to the ISO 17025 standard for its leading-edge mutation detection laboratory services designed to radically improve cancer diagnosis, monitoring, and treatment outcomes. Granted by SWEDAC, the Swedish Board for Accreditation and Conformity Assessment, ISO 17025 is an international quality management standard used to assess the competency of analytical laboratories. Genetic Analysis AS – The Norwegian Research Council approved Oslo-based Genetic Analysis’ grant application of 16 million kronor for its project developing new innovative microbiome-based diagnostics to be used to aid selection and treatment of IBD patients. The company plans to develop an easy-to-use, in vitro diagnostic test that profiles gut microbiota to predict disease progression and treatment regimes in patients with ulcerative colitis. Pilloxa – Swedish digital startup company Pilloxa today announced that it will collaborate with the Nordic office of Chiesi Global Rare Diseases to harness the capabilities of the Pilloxa platform to support the treatment journey for patients with nephropathic cystinosis across Scandinavia. Nephropathic cystinosis is an ultra-rare, multisystemic, genetic disorder characterized by the accumulation of the amino acid cystine in the lysosomes within the cells. Adlib – Toronto-based Adlib completed the SOC 2 Type I Service Organization Control (SOC 2) audit for its Content Intelligence platform in accord with the attestation standards established by the American Institute of Certified Public Accountants.