Opportunistic etiological agents causing lung infections: emerging need to transform lung-targeted delivery
New NTM Treatment Guidelines Recommend Insmed's ARIKAYCE® (amikacin ...
BRIDGEWATER, N.J., July 7, 2020 /PRNewswire/ -- Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, today announced that ARIKAYCE® (amikacin liposome inhalation suspension) has been included in the new international treatment guidelines for nontuberculous mycobacterial (NTM) lung disease, a chronic, debilitating condition that can cause severe, permanent damage to the lungs. The evidence-based guidelines, issued by the American Thoracic Society (ATS), European Respiratory Society (ERS), European Society of Clinical Microbiology and Infectious Diseases (ESCMID), and Infectious Diseases Society of America (IDSA), recommend the use of ARIKAYCE for the treatment of patients with refractory NTM lung disease caused by Mycobacterium avium complex (MAC) as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options.
Specifically, the guidelines strongly recommend the addition of ARIKAYCE to the standard treatment regimen for patients with MAC lung disease who have failed to convert to a negative sputum culture after at least six months of treatment.
The guidelines, published in Clinical Infectious Diseases and accessible here, are the globally recognized standard for the prevention, diagnosis, and treatment of NTM lung disease with the goal of providing the latest evidence-based guidance to improve patient care and outcomes. This is the first update to the guidelines in more than a decade, marking a significant milestone in the management of NTM lung disease.
"The inclusion of ARIKAYCE in the new NTM treatment guidelines highlights the critical role this innovative therapy can play in the management of patients with refractory MAC lung disease," said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. "We appreciate the dedication and hard work of the medical societies, physicians, and researchers who developed these guidelines, which will help improve patient care and outcomes for those battling this difficult-to-treat disease."
ARIKAYCE is the first and only therapy approved in the United States for the treatment of refractory NTM lung disease caused by MAC. ARIKAYCE received U.S. Food and Drug Administration (FDA) accelerated approval based on results of the Phase 3 CONVERT study, which demonstrated that once-daily ARIKAYCE, when combined with background regimen therapy, improved sputum culture conversion rates in patients with refractory NTM lung disease caused by MAC. In the study, the addition of ARIKAYCE to background regimen therapy eliminated evidence of NTM lung disease caused by MAC in sputum by Month 6 in 29% of patients, compared to 9% of patients on background regimen therapy alone.
About MAC Lung Disease
Mycobacterium avium complex (MAC) lung disease is a rare and serious disorder that can significantly increase morbidity and mortality. Patients with MAC lung disease can experience a range of symptoms that often worsen over time, including chronic cough, dyspnea, fatigue, fever, weight loss, and chest pain. In some cases, MAC lung disease can cause severe, even permanent damage to the lungs, and can be fatal. MAC lung disease is an emerging public health concern worldwide with significant unmet need.
About ARIKAYCE® (amikacin liposome inhalation suspension)
ARIKAYCE is the first and only FDA-approved therapy indicated for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. Current international treatment guidelines recommend the use of ARIKAYCE in patients with MAC lung disease who have failed standard therapy after at least six months of treatment. ARIKAYCE is a novel, inhaled, once-daily formulation of amikacin, an established antibiotic that was historically administered intravenously and associated with severe toxicity to hearing, balance, and kidney function. Insmed's proprietary PULMOVANCE™ liposomal technology enables the delivery of amikacin directly to the lungs, where liposomal amikacin is taken up by lung macrophages where the infection resides. This approach prolongs the release of amikacin in the lungs while limiting systemic exposure. ARIKAYCE is administered once daily using the Lamira® Nebulizer System manufactured by PARI Pharma GmbH (PARI).
About PARI Pharma and the Lamira® Nebulizer System
ARIKAYCE® (amikacin liposome inhalation suspension) is delivered by a novel inhalation device, the Lamira® Nebulizer System, developed by PARI. Lamira® is a quiet, portable nebulizer that enables efficient aerosolization of liquid medications, including liposomal formulations such as ARIKAYCE, via a vibrating, perforated membrane. Based on PARI's 100-year history working with aerosols, PARI is dedicated to advancing inhalation therapies by developing innovative delivery platforms and new pharmaceutical formulations that work together to improve patient care.
About CONVERT (INS-212)
CONVERT was a randomized, open-label, global Phase 3 study designed to confirm the sputum culture conversion results seen in Insmed's Phase 2 clinical study of ARIKAYCE in patients with refractory NTM lung disease caused by MAC. CONVERT was conducted in 18 countries at more than 125 sites. The primary efficacy endpoint was the proportion of patients who achieved sputum culture conversion at Month 6 in the ARIKAYCE plus GBT arm compared to the GBT-only arm. Patients who achieved sputum culture conversion by Month 6 continued in the CONVERT study for an additional 12 months of treatment following the first monthly negative sputum culture. The CONVERT study also evaluated the proportion of patients who maintained sputum culture conversion 3 months off all MAC treatment.
IMPORTANT SAFETY INFORMATION FOR ARIKAYCE IN THE U.S.
WARNING: RISK OF INCREASED RESPIRATORY ADVERSE REACTIONS ARIKAYCE has been associated with an increased risk of respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbation of underlying pulmonary disease that have led to hospitalizations in some cases.Hypersensitivity Pneumonitis has been reported with the use of ARIKAYCE in the clinical trials. Hypersensitivity pneumonitis (reported as allergic alveolitis, pneumonitis, interstitial lung disease, allergic reaction to ARIKAYCE) was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (3.1%) compared to patients treated with a background regimen alone (0%). Most patients with hypersensitivity pneumonitis discontinued treatment with ARIKAYCE and received treatment with corticosteroids. If hypersensitivity pneumonitis occurs, discontinue ARIKAYCE and manage patients as medically appropriate.
Hemoptysis has been reported with the use of ARIKAYCE in the clinical trials. Hemoptysis was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (17.9%) compared to patients treated with a background regimen alone (12.5%). If hemoptysis occurs, manage patients as medically appropriate.
Bronchospasm has been reported with the use of ARIKAYCE in the clinical trials. Bronchospasm (reported as asthma, bronchial hyperreactivity, bronchospasm, dyspnea, dyspnea exertional, prolonged expiration, throat tightness, wheezing) was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (28.7%) compared to patients treated with a background regimen alone (10.7%). If bronchospasm occurs during the use of ARIKAYCE, treat patients as medically appropriate.
Exacerbations of underlying pulmonary disease has been reported with the use of ARIKAYCE in the clinical trials. Exacerbations of underlying pulmonary disease (reported as chronic obstructive pulmonary disease (COPD), infective exacerbation of COPD, infective exacerbation of bronchiectasis) have been reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (14.8%) compared to patients treated with background regimen alone (9.8%). If exacerbations of underlying pulmonary disease occur during the use of ARIKAYCE, treat patients as medically appropriate.
Anaphylaxis and Hypersensitivity Reactions: Serious and potentially life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in patients taking ARIKAYCE. Signs and symptoms include acute onset of skin and mucosal tissue hypersensitivity reactions (hives, itching, flushing, swollen lips/tongue/uvula), respiratory difficulty (shortness of breath, wheezing, stridor, cough), gastrointestinal symptoms (nausea, vomiting, diarrhea, crampy abdominal pain), and cardiovascular signs and symptoms of anaphylaxis (tachycardia, low blood pressure, syncope, incontinence, dizziness). Before therapy with ARIKAYCE is instituted, evaluate for previous hypersensitivity reactions to aminoglycosides. If anaphylaxis or a hypersensitivity reaction occurs, discontinue ARIKAYCE and institute appropriate supportive measures.
Ototoxicity has been reported with the use of ARIKAYCE in the clinical trials. Ototoxicity (including deafness, dizziness, presyncope, tinnitus, and vertigo) were reported with a higher frequency in patients treated with ARIKAYCE plus background regimen (17%) compared to patients treated with background regimen alone (9.8%). This was primarily driven by tinnitus (7.6% in ARIKAYCE plus background regimen vs 0.9% in the background regimen alone arm) and dizziness (6.3% in ARIKAYCE plus background regimen vs 2.7% in the background regimen alone arm). Closely monitor patients with known or suspected auditory or vestibular dysfunction during treatment with ARIKAYCE. If ototoxicity occurs, manage patients as medically appropriate, including potentially discontinuing ARIKAYCE.
Nephrotoxicity was observed during the clinical trials of ARIKAYCE in patients with MAC lung disease but not at a higher frequency than background regimen alone. Nephrotoxicity has been associated with the aminoglycosides. Close monitoring of patients with known or suspected renal dysfunction may be needed when prescribing ARIKAYCE.
Neuromuscular Blockade: Patients with neuromuscular disorders were not enrolled in ARIKAYCE clinical trials. Patients with known or suspected neuromuscular disorders, such as myasthenia gravis, should be closely monitored since aminoglycosides may aggravate muscle weakness by blocking the release of acetylcholine at neuromuscular junctions.
Embryo-Fetal Toxicity: Aminoglycosides can cause fetal harm when administered to a pregnant woman. Aminoglycosides, including ARIKAYCE, may be associated with total, irreversible, bilateral congenital deafness in pediatric patients exposed in utero. Patients who use ARIKAYCE during pregnancy, or become pregnant while taking ARIKAYCE should be apprised of the potential hazard to the fetus.
Contraindications: ARIKAYCE is contraindicated in patients with known hypersensitivity to any aminoglycoside.
Most Common Adverse Reactions: The most common adverse reactions in Trial 1 at an incidence ≥5% for patients using ARIKAYCE plus background regimen compared to patients treated with background regimen alone were dysphonia (47% vs 1%), cough (39% vs 17%), bronchospasm (29% vs 11%), hemoptysis (18% vs 13%), ototoxicity (17% vs 10%), upper airway irritation (17% vs 2%), musculoskeletal pain (17% vs 8%), fatigue and asthenia (16% vs 10%), exacerbation of underlying pulmonary disease (15% vs 10%), diarrhea (13% vs 5%), nausea (12% vs 4%), pneumonia (10% vs 8%), headache (10% vs 5%), pyrexia (7% vs 5%), vomiting (7% vs 4%), rash (6% vs 2%), decreased weight (6% vs 1%), change in sputum (5% vs 1%), and chest discomfort (5% vs 3%).
Drug Interactions: Avoid concomitant use of ARIKAYCE with medications associated with neurotoxicity, nephrotoxicity, and ototoxicity. Some diuretics can enhance aminoglycoside toxicity by altering aminoglycoside concentrations in serum and tissue. Avoid concomitant use of ARIKAYCE with ethacrynic acid, furosemide, urea, or intravenous mannitol.
Overdosage: Adverse reactions specifically associated with overdose of ARIKAYCE have not been identified. Acute toxicity should be treated with immediate withdrawal of ARIKAYCE, and baseline tests of renal function should be undertaken. Hemodialysis may be helpful in removing amikacin from the body. In all cases of suspected overdosage, physicians should contact the Regional Poison Control Center for information about effective treatment.
U.S. INDICATION
LIMITED POPULATION: ARIKAYCE® is indicated in adults, who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. As only limited clinical safety and effectiveness data for ARIKAYCE are currently available, reserve ARIKAYCE for use in adults who have limited or no alternative treatment options. This drug is indicated for use in a limited and specific population of patients.
This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
Limitation of Use: ARIKAYCE has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of ARIKAYCE is not recommended for patients with non-refractory MAC lung disease.
Patients are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.Fda.Gov/medwatch, or call 1–800–FDA–1088. You can also call the Company at 1-844-4-INSMED.
Please see Full Prescribing Information.
About Insmed
Insmed Incorporated is a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases. Insmed's first commercial product, ARIKAYCE® (amikacin liposome inhalation suspension), is the first and only therapy approved in the United States for the treatment of refractory Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. MAC lung disease is a chronic, debilitating condition that can cause severe and permanent lung damage. Insmed's earlier-stage clinical pipeline includes brensocatib, a novel oral reversible inhibitor of dipeptidyl peptidase 1 with therapeutic potential in non-cystic fibrosis bronchiectasis and other inflammatory diseases, and treprostinil palmitil, an inhaled formulation of a treprostinil prodrug that may offer a differentiated product profile for rare pulmonary disorders, including pulmonary arterial hypertension. For more information, visit www.Insmed.Com.
Forward-looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. "Forward-looking statements," as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not historical facts and involve a number of risks and uncertainties. Words herein such as "may," "will," "should," "could," "would," "expects," "plans," "anticipates," "believes," "estimates," "projects," "predicts," "intends," "potential," "continues," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) may identify forward-looking statements.
The forward-looking statements in this press release are based upon the Company's current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which may cause the Company's actual results, performance and achievements and the timing of certain events to differ materially from the results, performance, achievements or timing discussed, projected, anticipated or indicated in any forward-looking statements. Such risks, uncertainties and other factors include, among others, the following: failure to successfully commercialize or maintain U.S. Approval for ARIKAYCE, the Company's only approved product; the risk that brensocatib does not prove effective or safe for patients in the STOP-COVID19 study; business or economic disruptions due to catastrophes or other events, including natural disasters or public health crises; impact of the novel coronavirus (COVID-19) pandemic and efforts to reduce its spread on our business, employees, including key personnel, patients, partners and suppliers; uncertainties in the degree of market acceptance of ARIKAYCE by physicians, patients, third-party payors and others in the healthcare community; the Company's inability to obtain full approval of ARIKAYCE from the FDA, including the risk that the Company will not timely and successfully complete the study to validate a PRO tool and complete the confirmatory post-marketing study required for full approval of ARIKAYCE; inability of the Company, PARI or the Company's other third party manufacturers to comply with regulatory requirements related to ARIKAYCE or the Lamira® Nebulizer System; the Company's inability to obtain adequate reimbursement from government or third-party payors for ARIKAYCE or acceptable prices for ARIKAYCE; development of unexpected safety or efficacy concerns related to ARIKAYCE or brensocatib; inaccuracies in the Company's estimates of the size of the potential markets for ARIKAYCE or brensocatib or in data the Company has used to identify physicians; expected rates of patient uptake, duration of expected treatment, or expected patient adherence or discontinuation rates; the Company's inability to create an effective direct sales and marketing infrastructure or to partner with third parties that offer such an infrastructure for distribution of ARIKAYCE; failure to obtain regulatory approval to expand ARIKAYCE's indication to a broader patient population; failure to successfully conduct future clinical trials for ARIKAYCE, brensocatib and the Company's other product candidates, including due to the Company's limited experience in conducting preclinical development activities and clinical trials necessary for regulatory approval and the Company's inability to enroll or retain sufficient patients to conduct and complete the trials or generate data necessary for regulatory approval; risks that the Company's clinical studies will be delayed or that serious side effects will be identified during drug development; failure to obtain, or delays in obtaining, regulatory approvals for ARIKAYCE outside the U.S. Or for the Company's product candidates in the U.S., Europe, Japan or other markets, including the United Kingdom as a result of its recent exit from the European Union; failure of third parties on which the Company is dependent to manufacture sufficient quantities of ARIKAYCE or the Company's product candidates for commercial or clinical needs, to conduct the Company's clinical trials, or to comply with laws and regulations that impact the Company's business or agreements with the Company; the Company's inability to attract and retain key personnel or to effectively manage the Company's growth; the Company's inability to adapt to its highly competitive and changing environment; the Company's inability to adequately protect its intellectual property rights or prevent disclosure of its trade secrets and other proprietary information and costs associated with litigation or other proceedings related to such matters; restrictions or other obligations imposed on the Company by its agreements related to ARIKAYCE or the Company's product candidates, including its license agreements with PARI and AstraZeneca AB, and failure of the Company to comply with its obligations under such agreements; the cost and potential reputational damage resulting from litigation to which the Company is or may become a party, including product liability claims; the Company's limited experience operating internationally; changes in laws and regulations applicable to the Company's business, including any pricing reform, and failure to comply with such laws and regulations; inability to repay the Company's existing indebtedness and uncertainties with respect to the Company's ability to access future capital; and delays in the execution of plans to build out an additional FDA-approved third-party manufacturing facility and unexpected expenses associated with those plans.
The Company may not actually achieve the results, plans, intentions or expectations indicated by the Company's forward-looking statements because, by their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future. For additional information about the risks and uncertainties that may affect the Company's business, please see the factors discussed in Item 1A, "Risk Factors," in the Company's Annual Report on Form 10-K for the year ended December 31, 2019, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2020 and any subsequent Company filings with the SEC.
The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date of this press release. The Company disclaims any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.
Contact:
Investors:
Argot PartnersLaura Perry or Heather Savelle(212) 600-1902Insmed@argotpartners.Com
Media:
Mandy FaheySenior Director, Corporate CommunicationsInsmed(732) 718-3621amanda.Fahey@insmed.Com
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SOURCE Insmed Incorporated
RedHill Biopharma Initiates Phase 3 Study Of RHB-204 For First-Line ...
TEL AVIV, Israel and RALEIGH, N.C., Nov. 20, 2020 /PRNewswire/ -- RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or "the Company"), a specialty biopharmaceutical company, today announced that it has initiated its Phase 3 study to evaluate the safety and efficacy of RHB-204 as a potential first-line, stand-alone, oral treatment of pulmonary nontuberculous mycobacteria (NTM) disease caused by Mycobacterium avium Complex (MAC) – a rare disease for which there is no FDA-approved first-line therapy.
"NTM is a debilitating disease that can cause scarring, fibrosis and the formation of cavities or pits in the lungs, which can lead to potentially fatal respiratory failure. People with existing lung conditions, such as bronchiectasis and those with COPD, are particularly susceptible," said Prof. Kevin Winthrop, MD, MPH, Professor of Infectious Diseases, Oregon Health & Science University, and study Principal Investigator. "NTM is notoriously resistant to most antibiotics and challenging to treat, and there is no FDA-approved first-line therapy for the approximately 110,000 cases of NTM infection in the U.S. This study of orally-administered RHB-204, if successful, represents an opportunity to make a breakthrough in managing NTM infections."
"Treatment of NTM disease requires multiple antibiotics and an extended treatment course due to the risk of development of resistance1," said Aida Bibliowicz, RedHill's Vice President of Clinical Affairs. "Many patients fail these types of therapies and more than half will have either recurring disease or a new infection after completing treatment2, making new treatment options for NTM an urgent need."
The multi-center, randomized, double-blind, two-part, placebo-controlled, parallel-group Phase 3 study will be conducted at up to 40 sites across the U.S. And aims to enroll 125 patients, randomized at a 3:2 ratio to receive either RHB-204 or placebo. The study is designed to evaluate the safety and efficacy of RHB-204 in patients with symptomatic Mycobacterium avium Complex (MAC) lung disease. Study endpoints include sputum culture conversion at month six of treatment with RHB-204, compared to placebo and patient-reported outcomes, including improvements in physical functioning, respiratory symptoms and fatigue. Following this assessment (part one of the study), patients may be eligible to continue double-blinded treatment for up to 16 months (part two). Sustainability of clinical benefit and durability of microbiological response will be assessed at month 16 and again three months after treatment completion.
RHB-204 was recently granted Orphan Drug designation, extending U.S. Market exclusivity for RHB-204 by an additional seven years, for a potential total of 12 years upon FDA approval. RHB-204 had also previously been granted a Qualified Infectious Disease Product (QIDP) designation by the FDA, providing eligibility for Fast-Track development, NDA Priority Review and a five-year extension of U.S. Market exclusivity, if approved.
The Phase 3 study of RHB-204 is registered on www.ClinicalTrials.Gov, a web-based service by the U.S. National Institute of Health, which provides public access to information on publicly and privately supported clinical studies.
About Pulmonary Nontuberculous Mycobacteria (NTM) Disease
Pulmonary nontuberculous mycobacteria (NTM) disease is a chronic and debilitating lung disease caused by ubiquitous environmental bacteria found in soil, as well as natural and engineered water systems. The most common NTM symptoms include fever, weight loss, chest pain, and blood in sputum3. Pulmonary NTM disease can lead to recurring cases of bronchitis and pneumonia and can, in some cases, lead to respiratory failure4. Although rare, the incidence and prevalence of pulmonary NTM disease are increasing in many areas of the world5. There were an estimated 110,000 pulmonary NTM disease patients in the U.S. In 20176. Pulmonary manifestations account for 80-90% of all NTM-associated diseases7, and approximately 80% of pulmonary NTM disease are caused by Mycobacterium avium Complex (MAC)8.
About RHB-204
RHB-204 is a proprietary, fixed-dose oral capsule containing a combination of clarithromycin, rifabutin, and clofazimine, developed for the treatment of pulmonary NTM disease caused by Mycobacterium avium Complex (MAC). RHB-204 was granted both FDA Orphan Drug designation for the treatment of NTM disease and QIDP Designation under the Generating Antibiotic Incentives Now Act (GAIN Act), extending U.S. Market exclusivity for RHB-204 to a potential total of 12 years to be granted at the time of FDA approval. RHB-204 is also covered by U.S. Patents which extend patent protection until 2029 and a pending U.S. Patent application which, if allowed, could extend RHB-204 patent protection until 2041.
About RedHill Biopharma
RedHill Biopharma Ltd. (Nasdaq: RDHL) is a specialty biopharmaceutical company primarily focused on gastrointestinal and infectious diseases. RedHill promotes the gastrointestinal drugs, Movantik® for opioid-induced constipation in adults9, Talicia®for the treatment of Helicobacter pylori (H. Pylori) infection in adults10, and Aemcolo® for the treatment of travelers' diarrhea in adults11. RedHill's key clinical late-stage development programs include: (i) RHB-204, with an ongoing Phase 3 study for pulmonary nontuberculous mycobacteria (NTM) disease; (ii) opaganib (Yeliva®), a first-in-class SK2 selective inhibitor targeting multiple indications with a Phase 2/3 program for COVID-19 and Phase 2 studies for prostate cancer and cholangiocarcinoma ongoing; (iii) RHB-104, with positive results from a first Phase 3 study for Crohn's disease; (iv) RHB-102 (Bekinda®), with positive results from a Phase 3 study for acute gastroenteritis and gastritis and positive results from a Phase 2 study for IBS-D; (v) RHB-107 (upamostat), a Phase 2-stage serine protease inhibitor with a planned Phase 2/3 study in symptomatic COVID-19 and targeting multiple other cancer and inflammatory gastrointestinal diseases; and (vi) RHB-106, an encapsulated bowel preparation. More information about the Company is available at www.Redhillbio.Com.
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements may be preceded by the words "intends," "may," "will," "plans," "expects," "anticipates," "projects," "predicts," "estimates," "aims," "believes," "hopes," "potential" or similar words. Forward-looking statements are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company's control and cannot be predicted or quantified, and consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation; the risk that the Company will not succeed to complete the patient recruitment; the risk that the U.S. Phase 3 clinical study evaluating RHB-204 will not be successful or, if successful, will not suffice for regulatory marketing approval without the need for additional clinical and/or other studies; as well as risks and uncertainties associated with (i) the initiation, timing, progress and results of the Company's research, manufacturing, pre-clinical studies, clinical trials, and other therapeutic candidate development efforts, and the timing of the commercial launch of its commercial products and ones it may acquire or develop in the future; (ii) the Company's ability to advance its therapeutic candidates into clinical trials or to successfully complete its pre-clinical studies or clinical trials or the development of a commercial companion diagnostic for the detection of MAP; (iii) the extent and number and type of additional studies that the Company may be required to conduct and the Company's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings, approvals and feedback; (iv) the manufacturing, clinical development, commercialization, and market acceptance of the Company's therapeutic candidates and Talicia®; (v) the Company's ability to successfully commercialize and promote Talicia®, and Aemcolo® and Movantik®; (vi) the Company's ability to establish and maintain corporate collaborations; (vii) the Company's ability to acquire products approved for marketing in the U.S. That achieve commercial success and build its own marketing and commercialization capabilities; (viii) the interpretation of the properties and characteristics of the Company's therapeutic candidates and the results obtained with its therapeutic candidates in research, pre-clinical studies or clinical trials; (ix) the implementation of the Company's business model, strategic plans for its business and therapeutic candidates; (x) the scope of protection the Company is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; (xi) parties from whom the Company licenses its intellectual property defaulting in their obligations to the Company; (xii) estimates of the Company's expenses, future revenues, capital requirements and needs for additional financing; (xiii) the effect of patients suffering adverse experiences using investigative drugs under the Company's Expanded Access Program; (xiv) competition from other companies and technologies within the Company's industry; and (xv) the hiring and employment commencement date of executive managers. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the Securities and Exchange Commission (SEC), including the Company's Annual Report on Form 20-F filed with the SEC on March 4, 2020. All forward-looking statements included in this press release are made only as of the date of this press release. The Company assumes no obligation to update any written or oral forward-looking statement, whether as a result of new information, future events or otherwise unless required by law.
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SOURCE RedHill Biopharma Ltd.
Treating Nontuberculous Mycobacteria (NTM) - News-Medical.net
Nontuberculous mycobacteria (NTM) are mycobacteriaceae species that can cause the onset of illness in humans. NTM are present naturally in the soil and air. But NTM are not the causative agents of tuberculosis. These bacteria have been classified as nontuberculosis to set them apart from the tuberculosis-causing bacteria, also known as the Mycobacterium tuberculosis complex.
Among the more common NTM that must be therapeutically addressed are M. Avium, M. Intracellulare, M. Kansasii, M. Abscessus, M. Chelonae, M. Fortuitum, M. Terrae, M. Xenopi, and M. Simiae.
Treatment is a ChallengeHow to treat a NTM-related condition is not be a simple, straightforward decision for a clinician to make. Most often, treatment will involve the administration of a variety of antibiotics. But NTM have antibiotic resistance. Moreover, treating NTM with only one drug might actually cause the condition to become more antibiotic-resistant than if several drugs are administered.
A successful treatment will necessitate the use of two or three therapeutics. Therapies recommended by the American Thoracic Society to treat pulmonary NTM include: azithromycin, rifampin, ethambutol, and streptomycin. The precise drugs to be administered will be determined by the exact nature of the bacteria, the extent of infection, and which drugs the bacteria may be sensitive to. Therapy must continue until cultures of the respiratory system yield negative results for one year.
Specific regimens will vary. The susceptibility of the bacteria to specific drugs is one determining issue. The exact nature of the treatment will also be influenced by the patient's age, overall health, the specific symptoms, personal preferences, and if they are allergic to any of the drugs.
The American Thoracic Society and Infectious Diseases Society of America offer extensive treatment plans. Their drug plans cover frequency, duration, dosage, toxicity, and preventive treatment. Distinctions are made as to how to deal with different types of bacteria, as well as how to address slow- and fast-growing infections.
For example, in order to treat Mycobacterium avium Complex pulmonary disease and M. Kansasii, which represent the most common causes of lung-based NTM disease, a patient must receive three drugs, depending on the severity of the condition. These might be administered each day or on three days of the week. On the other hand, several months of treatment with intravenously-administered drugs, oral, and inhaled antibiotic drugs are needed for those suffering from M. Abscessus. Health outcomes are sometimes not good for patients, and surgery may be required to take out damaged areas of the lung.
Also helpful for infected individuals would be to force mucus from their lungs by applying physical therapy to the chest, undertaking aerobic exercise, or administering therapies as a mist inhaled into the lungs. It also would be important for patients to maintain a healthy weight and consume healthy and nutritious products. Regardless of whether a patient is treated or not, they must be monitored to determine if the condition becomes worse.
NTM therapeutics may cause side effects that can be cause for concern. For instance, they may be poorly tolerated among certain populations. Many therapies are costly, and the expense grows the longer the patient is required to remain on the treatment regimen. Despite these, it is necessary to balance the risks against the benefits for each individual, and to consider the outcome if the condition is not addressed and treated, especially if the symptoms are severe.
Avoiding ReinfectionIt is possible to become infected with NTM either by breathing in air by drinking water so, to avoid future infections, individuals should become more aware of where the bacteria might exist in the environment. Avoiding hot tubs and exposure to certain bodies of water and soil will help lessen the likelihood of reinfection, as would boiling tap water intended for drinking.
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