RESEARCH

Orsini Specialty Pharmacy Expands Collaborates with Alnylam® Pharmaceuticals as a Limited Distribution Partner for Oxlumo™

Orsini | December 04, 2020

Orsini Specialty Pharmacy, an innovator in uncommon illnesses and quality treatments, reported today that it has been chosen by Alnylam® Pharmaceuticals as a restricted appropriation accomplice for OXLUMO™ (lumasiran). OXLUMO is the solitary treatment affirmed for essential hyperoxaluria type 1 (PH1) to bring down urinary oxalate levels in pediatric and grown-up patients.

PH1 is a super uncommon hereditary sickness described by the overproduction of oxalate, a side-effect that normally is dispensed with by the kidneys and discharged in the pee. The overabundance creation of oxalate brings about the affidavit of calcium oxalate gems in the kidneys and urinary plot and can prompt the development of agonizing and repetitive kidney stones, nephrocalcinosis, movement to kidney disappointment, and foundational organ brokenness. It is assessed that PH1 influences one to three people for each million in the United States and Europe.

"We are satisfied to grow our organization with Alnylam Pharmaceuticals and add OXLUMO to the treatments we accommodate the treatment of super uncommon hereditary conditions," said Mike Fieri, Orsini Chief Executive Officer. "Our devoted OXLUMO Care Team is focused on offering caring help and individualized administrations to help individuals with PH1 and their families all through their excursion and assist them with exploring through the complexities of our medical services framework."

Spotlight

Pediatric populations represent the most diverse patient
groups in all of medicine. Factors such as individual
preferences, physiology, and dosage considerations all serve to complicate drug formulation aimed at pediatric care.

Spotlight

Pediatric populations represent the most diverse patient
groups in all of medicine. Factors such as individual
preferences, physiology, and dosage considerations all serve to complicate drug formulation aimed at pediatric care.

Related News

BUSINESS INSIGHTS, PHARMA TECH

Innate Pharma Announces Preclinical Data Publication in Nature Biotechnology

Innate Pharma | January 17, 2023

On January 16, 2023, Innate Pharma SA announced the publication of preclinical findings demonstrating the control of acute myeloid leukemia (AML) cells by a trifunctional NKp46-CD16a-NK cell engager (NKCE) targeting CD123 in the scientific journal Nature Biotechnology. Innate and Sanofi conducted the experiments, which were published in Nature Biotechnology on January 12, 2023. This study demonstrates that CD64 expression on AML blasts imparts resistance to anti-CD123 antibody-dependent cell cytotoxicity (ADCC) and that redirecting NK cells against cancer targets by binding to CD16a and NKp46 circumvents this resistance. Moreover, through their binding to NKp46, CD123-NKCE primarily target NK cells and have substantial antitumor activity against primary AML blasts; they stimulate NK cell activation and cytokine secretion exclusively in AML cells' presence. In vivo, its antitumor activity observed in a mouse tumor model surpasses that of the anti-CD123 antibody used as the comparator. Furthermore, in vitro in human peripheral blood mononuclear cells and in vivo in nonhuman primates, the efficacy of CD123-NKCE was associated with the induction of low pro-inflammatory cytokine release and the absence of toxicity. These results support CD123-NKCE clinical development. IPH6101/SAR'579 (SAR443579), the first NKp46/CD16-based CD123-targeted ANKETTM NK cell engager, is currently being evaluated in a Phase 1/2 clinical trial by Sanofi in patients with relapsed or refractory acute myeloid leukemia (R/R AML), B-cell acute lymphoblastic leukemia (B-ALL), or high-risk myelodysplastic syndrome (HR-MDS). About Innate Pharma Headquartered in Marseille (France), Innate Pharma SA is a global, clinical-stage oncology-focused biotech firm dedicated to enhancing patient treatment and clinical outcomes using therapeutic antibodies that employ the immune system to combat cancer. Its extensive antibody pipeline has numerous potentially first-in-class clinical and preclinical candidates for cancers with a high unmet medical need. In addition, Innate has been a pioneer in the study of natural killer cell (NK cell) biology and developed its expertise in tumor antigens, tumor microenvironment, and antibody engineering. This innovative strategy has resulted in a varied proprietary portfolio and substantial alliances with biopharmaceutical industry leaders, including Sanofi, Bristol-Myers Squibb, Novo Nordisk A/S, and a multi-product collaboration with AstraZeneca.

Read More

BUSINESS INSIGHTS, PHARMA TECH

Rallybio Corporation and EyePoint Announce Research Cooperation

EyePoint Pharmaceuticals | February 28, 2023

On February 27, 2023, EyePoint Pharmaceuticals, Inc., a leading pharmaceutical firm committed to the development and commercialization of therapeutics to help improve the lives of patients with severe eye disorders and Rallybio Corporation, a clinical-stage biotechnology company dedicated to identifying and accelerating the development of life-transforming therapeutics for patients with severe and rare diseases, announced a research collaboration to explore the sustained delivery of Rallybio's complement component 5 (C5) inhibitor for the treatment of geographic atrophy, an advanced form of age-related macular degeneration that can cause irreversible vision loss. The research will use EyePoint's proprietary Durasert® technology, which is designed for sustained intraocular drug delivery. Approximately one million individuals in the United States suffer from geographic atrophy, which is characterized by atrophic lesions in the central region of the macula. Geographic atrophy can cause irreversible vision loss and can lead to legal blindness. Currently, geographic atrophy treatments are limited in choice and duration of action, leaving a significant unmet need for safe and effective treatment options. Under the terms of the research partnership, EyePoint and Rallybio will assess and explore the viability of utilizing Rallybio's C5 inhibitor in EyePoint's Durasert technology, with the intention to increase the collaboration upon mutual agreement following the evaluation. EyePoint Pharmaceuticals' M.D., President and Chief Operating Officer Jay Duker commented, "We are excited to begin this research collaboration to explore the combination of Rallybio's C5 inhibitor with our bioerodible Durasert sustained release drug delivery technology to develop a potential long-acting treatment for geographic atrophy," He further emphasized, "Geographic atrophy associated with dry macular degeneration is a devastating eye disease, and the inhibition of complement is a proven treatment pathway. We hope to leverage our Durasert technology in this collaboration to create a potential best-in-class, long-acting intravitreal insert, which we believe could provide a more desirable option for patients given that the existing approved therapy is injected every one to two months." (Source – Business Wire) About EyePoint Pharmaceuticals EyePoint Pharmaceuticals is a leading pharmaceutical company that develops and commercializes therapeutic products to help patients suffering from serious eye disorders. Headquartered in Watertown, Massachusetts, it is committed to using its innovative drug delivery platform to improve patients' quality of life. The company's pipeline utilizes its proprietary Durasert technology, which enables sustained intraocular drug delivery. Its current pipeline includes EYP-1901, a potential six-month intravitreal anti-VEGF treatment aimed at treating wet age-related macular degeneration. Durasert technology has been successfully used across four FDA-approved products, including YUTIQ®, which treats chronic non-infectious uveitis affecting the posterior segment of the eye.

Read More

BUSINESS INSIGHTS, PHARMA TECH

Koneksa Starts Trial Comparing At-home Mobile Spirometry to In-clinic Spirometry

Koneksa | January 18, 2023

On January 17, 2023, Koneksa, a healthcare technology company that develops evidence-based validated digital biomarkers, announced the beginning of a clinical study that will compare the treatment effect of at-home mobile spirometry using digital biomarkers versus in-clinic spirometry in patients who have moderate asthma and are receiving long-acting beta-agonist (LABA) treatment. The LEARN study is a single-arm interventional trial that compares the identification of treatment effects in people with moderate asthma using at-home mobile spirometry by utilizing an ultrasonic spirometer and a smartphone compared to in-clinic spirometry. Up to 60 people with mild, uncontrolled asthma who already use inhaled corticosteroids (ICS) will take part in the study. Besides, it will also cover those who need to take LABA medication for a six-week treatment period. In-clinic spirometry can be performed at home with the same or higher accuracy compared to in-clinic and potentially less strain on the patient. The study will analyze the LABA treatment impact in clinic and mobile spirometry along with assessing the association between different readouts of mobile spirometry, in addition to evaluating asthma control as determined by the ACQ-6, a patient-reported outcome questionnaire. Other goals include assessing patient and site-specific satisfaction with mobile spirometry technology. Chris Benko, CEO & Founder, Koneksa, stated, “This study builds on Koneksa's prior work demonstrating that mobile spirometry has the potential to detect clinically meaningful changes in very small cohorts of asthma patients on standard of care.” He further added, “The LEARN study will evaluate whether at-home mobile spirometry can detect treatment effects of a known positive control (LABA) in a significantly smaller population than would traditionally be assumed for an in-clinic proof-of-concept study.” (Source – Business Wire) About Koneksa Based out of New York, Koneksa is a patient-centric digital biomarker firm that develops end-to-end solutions for remotely obtained clinical data to expedite clinical research and guide decision-making in drug development and market strategy for the pharmaceutical and biotechnology industries. It seeks to revolutionize effect detection in clinical research by providing integrated solutions for efficient trial designs that yield more meaningful data. Its evidence-based biomarker solutions allow for more efficient clinical trial designs, allowing novel medicines to reach patients sooner.

Read More