Inceptua SA

While antimicrobial resistance is on the rise, a greater number of people still succumb to infections caused by drug-sensitive bacteria. The study reveals that ongoing development of drug resistance is unavoidable, driven by the large populations of pathogens and their exposure to antibiotics. Furthermore, the researchers say, opportunistic pathogens will continue to endanger patients with weakened immune systems, even if they are susceptible to existing antibiotics according to clinical microbiology tests. These issues are interconnected and are likely to intensify as the human population ages, becomes more densely packed, and faces disruptions like war, extreme weather events, or a decline in living standards. Now Inceptua Group, a global pharmaceutical service company, has announced the expansion of its Early Access Program for cefiderocol to include eligible patients in Argentina, Chile, Colombia, Guatemala, Mexico, and Panama. This initiative is in partnership with Shionogi & Co. Ltd. and approved by local regulatory authorities. Shionogi will supply US-labeled cefiderocol through the Inceptua Early Access Program on a case-by-case basis to patients outside of clinical trials. Serious gram-negative bacterial infections “Inceptua is pleased to announce the expansion of this Early Access Program to Latin America to allow physicians the opportunity to access this medicine for use with appropriate patients with certain serious gram-negative bacterial infections,” says Stuart Bell, executive vice president, Inceptua Early Access. googletag.cmd.push(function () { googletag.display('text-ad1'); }); Cefiderocol is a small molecule siderophore cephalosporin antibiotic developed by Shionogi for treating serious gram-negative infections. It penetrates the outer cell membrane of gram-negative pathogens by binding to ferric iron and being actively transported into bacterial cells. This allows it to reach high concentrations in the periplasmic space, where it inhibits cell wall synthesis. While cefiderocol is not yet approved for commercial use in Latin America, it has received approval from the US FDA in 2019, the European Medicines Agency (EMA) in 2020, and the Japanese Ministry of Health, Labor, and Welfare in 2023. Inceptua and Shionogi have collaborated since 2020 to make cefiderocol available through an Early Access program, initially starting in Europe and subsequently expanding into Canada and the Asia Pacific region. To date, nearly 1,000 patients have accessed cefiderocol through this program. Making medicines available for those with limited options Early Access Programs, also known as expanded access, managed access, compassionate use, and named patient supply, provide a compliant route for making medicines available to patients who either have limited or no alternative treatment options or have exhausted all other available treatments in their country of residence. In the US, cefiderocol is indicated for patients 18 years and older for treating complicated urinary tract infections (cUTIs), including pyelonephritis, and hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP), caused by susceptible gram-negative microorganisms such as Escherichia coli, Klebsiella pneumoniae, and Pseudomonas aeruginosa, among others. Inceptua Early Access offers strategic advice, design, facilitation, and implementation of global early access programs, distributing unlicensed and other medicines worldwide. Partnering with pharma and biotech companies, it has global reach, allowing patients access to potentially life-saving medicines. It has offices in eight countries and a global warehousing and distribution network and continues bringing promising medicines to patients. Inceptua Clinical Trial Supply provides clinical trial supply services, including comparator sourcing, packaging, labeling, storage, and distribution. With more than 25 years of experience and global operations across Europe, North America, and Asia, Inceptua serves life science companies of all sizes.

The partnership, announced today (January 18) will make NITYR (nitisinone) tablets available for eligible patients in the UK with Hereditary Tyrosinemia Type 1 (HT-1) and Alkaptonuria (AKU). A Free Goods Programme is a compliant way for medicines that are not approved in their country of intended use to be made available for patients who either have no alternative treatment option or have exhausted all other treatment options available in their country of residence. Stuart Bell, senior vice president, Inceptua early access said: “Inceptua is proud to partner with Cycle in this Free Goods Programme to allow access to NITYR to a broader number of countries and patients. Cycle’s ethos around medicine provision aligns very closely to those of Inceptua’s, and we look forward to supporting patients with no other treatment options for these devastating diseases.” Rare genetic condition Ht-1 is a very rare genetic condition. It prevents the body from breaking down a substance called tyrosine found in food. This leads to the build-up of toxic levels of substances in the blood. If left untreated, TYR1 can damage the liver, kidneys, and the nervous system. The NHS describes AKU, or black urine disease, as a very rare inherited disorder that prevents the body fully breaking down two protein building blocks (amino acids) called tyrosine and phenylalanine. It results in a build-up of a chemical called homogentisic acid in the body. This can turn urine and parts of the body a dark color and lead to a range of health problems over time. googletag.cmd.push(function () { googletag.display('text-ad1'); }); Cycle has an existing Free Goods Program with patients with HT-1 in India, Bangladesh, Pakistan, and Sudan. Inceptua will support Cycle through the expansion of the Free Goods Programme to include further countries where NITYR is not commercially available, or where the local healthcare system is unable to afford this medication. Countries in scope of the NITYR Free Goods Programme include Argentina, Bangladesh, Brazil, Chile, Colombia, Dominican Republic, Jordan, Mexico, Nigeria, Pakistan, Peru, Tunisia, and Turkey. Cycle says it is committed to supporting patients on the Free Goods Programme for the duration of their lifetime. Proud and humbled James Harrison, CEO of Cycle Pharmaceuticals said: “Right from the beginning of Cycle’s story in 2012, we made a commitment to provide a Free Goods Programme for rare disease patients that cannot otherwise access our medicines.  The whole Cycle team is extremely proud and humbled to be able to expand the NITYR Free Goods Programme. It really matters.” In its research, the companies found that HT-1 affects about 1 in 100,000 individuals worldwide, whilst AKU affects 1 in 250,000 to 1 million people worldwide. NITYR (nitisinone) tablets are a competitive inhibitor of 4-hydroxyphenyl-pyruvate dioxygenase indicated for the treatment of adult and pediatric patients with HT-1 in combination with dietary restriction of tyrosine and phenylalanine. NITYR has been approved for use by the FDA, Health Canada, EMA, MHRA and TGA.

SOUTH SAN FRANCISCO, Calif., July 14, 2022 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced the initiation of an expanded access program (EAP) for voxelotor in Brazil for the treatment of sickle cell disease (SCD) in adult and pediatric patients 12 years of age and older who cannot satisfactorily be treated with a medicine authorized for use in Brazil. ANVISA, the Brazilian Health Regulatory Agency, has authorized the voxelotor EAP, which is actively enrolling at clinical sites around the country. “There are up to 100,000 people estimated to have sickle cell disease in Brazil, yet there are no currently approved treatments that directly address the underlying cause of the disease,” said Kim Smith-Whitley, M.D., executive vice president and head of research and development at GBT. “As part of our deep, ongoing commitment to patient access worldwide, we are pleased to initiate this expanded access program for eligible patients in Brazil who may benefit from voxelotor, which we believe can potentially modify the course of this debilitating disease.” A first-in-class oral, once-daily therapy, voxelotor directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. The sickling process causes anemia and hemolysis (low hemoglobin due to red blood cell destruction), which impairs adequate oxygen delivery to tissues and organs in the body. Voxelotor is not currently approved for marketing by ANVISA in Brazil. An expanded access program is a mechanism to make medicines available pre-approval upon request by a physician for eligible patients with no alternative treatment option. ANVISA’s authorization of an EAP is based on the following criteria: The product is intended for patients with a serious, debilitating, and/or life-threatening disease.There is no satisfactory therapeutic alternative with products registered in Brazil.The authorization to use the product is delivered based on the request, and only under the responsibility of the prescribing physician, as it is expected to deliver a significant benefit to the patient. Under the voxelotor EAP, voxelotor will be supplied free of charge for as long as the patient requires treatment. This program is being implemented in partnership with and administered by Inceptua Group’s Medicines Access division, which has expertise in the strategy, design, and operational implementation of pre-approval access programs that make pharmaceutical products available to patients as appropriate in multiple countries around the world. About Sickle Cell Disease It is estimated that more than 100,000 people in the United States,1 52,000 people in Europe,2 up to 100,000 people in Brazil,3 and millions of people throughout the world have sickle cell disease (SCD).1 SCD occurs particularly among those whose ancestors are from sub-Saharan Africa, though it also occurs in people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.1 SCD is a lifelong inherited blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.4 Due to a genetic mutation, individuals with SCD form abnormal hemoglobin known as sickle hemoglobin. When sickle hemoglobin becomes deoxygenated, it polymerizes to form rods, which deforms the red blood cells into sickled – crescent-shaped, rigid – cells.4,5,6 The recurrent sickling process causes destruction of the red blood cells, hemolysis and anemia (low hemoglobin due to red blood cell destruction), which drives vascular inflammation contributing to blockages in capillaries and small blood vessels (vaso-occlusion) that impede the flow of blood and oxygen delivery throughout the body. Episodes of painful vascular occlusions are commonly referred to as vaso-occlusive crises (VOCs). The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.5,6,7,8,9 Complications of SCD begin in early childhood and can include neurocognitive impairment, acute chest syndrome, and silent and overt stroke, among other serious issues.10 Early intervention and treatment of SCD have shown potential to modify the course of this disease, reduce symptoms and events, prevent long-term organ damage, and extend life expectancy.5 About VoxelotorVoxelotor is an oral, once-daily therapy for patients with sickle cell disease (SCD). Voxelotor works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, voxelotor inhibits sickle hemoglobin polymerization and the resultant sickling and destruction of red blood cells leading to hemolysis and anemia, which are primary pathologies faced by every single person living with SCD. Through addressing hemolysis and anemia and improving oxygen delivery throughout the body, GBT believes that voxelotor has the potential to modify the course of SCD. In November 2019, the U.S. Food and Drug Administration (FDA) granted accelerated approval for voxelotor tablets, under the brand name Oxbryta®, for the treatment of SCD in adults and children 12 years of age and older, and in December 2021, the FDA expanded the approved use of Oxbryta for the treatment of SCD in patients 4 years of age and older in the United States.10 As a condition of accelerated approval for patients ages 4 and older in the United States, GBT is studying Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to assess the ability of the therapy to decrease stroke risk in children 2 to 14 years of age. In recognition of the critical need for new SCD treatments, the FDA granted Oxbryta Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations for the treatment of patients with SCD. Additionally, Oxbryta received the prestigious 2021 Prix Galien USA award for “Best Biotechnology Product” from The Galien Foundation. Please click here for Important Safety Information and full Prescribing Information, including Patient Information for Oxbryta in the U.S. About Global Blood TherapeuticsGlobal Blood Therapeutics (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities, starting with sickle cell disease (SCD). Founded in 2011, GBT is delivering on its goal to transform the treatment and care of SCD, a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta (voxelotor), the first FDA-approved medicine that directly inhibits sickle hemoglobin (HbS) polymerization, the root cause of red blood cell sickling in SCD. GBT is also advancing its pipeline program in SCD with inclacumab, a P-selectin inhibitor in Phase 3 development to address pain crises associated with the disease, and GBT021601 (GBT601), the company’s next generation HbS polymerization inhibitor. In addition, GBT’s drug discovery teams are working on new targets to develop the next wave of potential treatments for SCD. To learn more, please visit www.gbt.com and follow the company on Twitter @GBT_news. Forward-Looking Statements Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995, including statements containing the words “will,” “anticipates,” “plans,” “believes,” “forecast,” “estimates,” “expects” and “intends,” or similar expressions. These forward-looking statements are based on GBT’s current expectations and actual results could differ materially. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. GBT intends these forward-looking statements, including statements regarding GBT’s priorities, dedication, commitment, focus, goals, mission, vision and positioning; safety, efficacy and mechanism of action of Oxbryta (or voxelotor) and other product characteristics; commercialization, delivery, availability, use and commercial and medical potential of Oxbryta; the expanded access program for Oxbryta in Brazil, including the potential availability, enrollment, use, impact and related expectations; ongoing and planned studies, clinical trials and registries, and related protocols, activities, timing, and other expectations; regulatory submission, review and approval; impacting the treatment, course and care of SCD; safety, efficacy, mechanism of action, advancement and potential of GBT’s drug candidates and pipeline; and working on new targets and discovering, developing and delivering treatments, to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act, and GBT makes this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect GBT’s current views about its plans, intentions, expectations, strategies and prospects, which are based on the information currently available to the company and on assumptions the company has made. GBT can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved, and, furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond GBT’s control, including, without limitation, risks and uncertainties relating to the COVID-19 pandemic, including the extent and duration of the impact on GBT’s business, including commercialization activities, regulatory efforts, research and development, corporate development activities and operating results, which will depend on future developments that are highly uncertain and cannot be accurately predicted, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the U.S. and in other countries, and the effectiveness of actions taken globally to contain and treat the disease; the risks that GBT is continuing to establish its commercialization capabilities and may not be able to successfully commercialize Oxbryta; risks associated with GBT’s dependence on third parties for research, development, manufacture, distribution and commercialization activities; government and third-party payer actions, including those relating to reimbursement and pricing; risks and uncertainties relating to competitive treatments and other changes that may limit demand for Oxbryta; the risks regulatory authorities may require additional studies or data to support continued commercialization of Oxbryta; the risks that drug-related adverse events may be observed during commercialization or clinical development; data and results may not meet regulatory requirements or otherwise be sufficient for further development, regulatory review or approval; compliance with obligations under the Pharmakon loan; and the timing and progress of activities under GBT’s collaboration, license and distribution agreements; along with those risks set forth in GBT’s Annual Report on Form 10-K for the fiscal year ended December 31, 2021, and in GBT’s most recent Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission, as well as discussions of potential risks, uncertainties and other important factors in GBT’s subsequent filings with the U.S. Securities and Exchange Commission. Except as required by law, GBT assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. References Centers for Disease Control and Prevention. Sickle Cell Disease Data and Statistics (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed June 7, 2022.European Medicines Agency. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. Accessed June 12, 2022.Ministério da Saúde (Brasil), Protocolo Clínico e Diretrizes Terapêuticas da Doença Falciforme, Feb. 19, 2018National Heart, Lung, and Blood Institute. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Accessed February 23, 2022.Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.Rees DC, et al. Lancet. 2010;376(9757):2018-2031.Kato GJ, et al. J Clin Invest. 2017;127(3):750-760. Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23.Nader E, et al. Front Immunol. 2020 Mar 13;11:454.Kanter J, et al. Blood Rev. 2013 Nov;27(6):279-87. Contact:Steven Immergut (media)+1 650-410-3258simmergut@gbt.com Courtney Roberts (investors)+1 650-351-7881croberts@gbt.com