RESEARCH

Rhythm Pharmaceuticals Receives FDA Approval for obesity med targeting genetic defects

Rhythm | December 01, 2020

Stoutness is a significant issue around the world, in any case, for a small amount of patients, hereditary changes instead of propensities are driving the sickness. With its new FDA endorsement for Imcivree, Rhythm Pharmaceuticals is venturing out treating those cases.

For a subset of heftiness patients, hereditary transformations leave them with an "voracious appetite," Rhythm CEO David Meeker, M.D., said in a meeting. Beginning stage hereditary illnesses can leave patients incapable to control corpulence with diet and exercise, and, presently, Rhythm has scored the main FDA endorsement for a focused on medication pointed toward treating them.

Imcivree, once known as setmelanotide, scored a FDA gesture to treat patients who have varieties of the POMC, PCSK1 or LEPR qualities. The medication focuses on the MC4R pathway answerable for directing yearning, and the three qualities remembered for the underlying endorsement are only an initial step, Meeker said. The organization built up a test that takes a gander at around 100 hereditary transformations, and it's trying the medication in other hereditary changes with eyes on expected endorsements down the line.

With obesity, “you can see the forest,” Meeker said, because the problem is everywhere. The challenge is finding patients who could have an underlying genetic mutations causing excessive hunger, he said, noting that “testing becomes key.”

Most people, and most doctors, still think of obesity as a “calories in, calories out,” problem, said Michael Dedekian, medical director of pediatric specialty care at Barbara Bush Children’s Hospital in Maine. Very quickly, though, the medical community’s understanding of obesity is deepening, he said. And, as that’s happening, it’s becoming clear that genetic defects can “wildly disregulate” patients’ hunger. 

With the approval and new knowledge about genetic defects, Dedekian believes the medical community is entering a “whole new era of obesity treatment." Over time, he has no doubt awareness of the genetic mutations driving some cases will increase. When abnormal hunger presents itself in young children, doctors should strongly consider a genetic test, Dedekian said.

Rhythm isn’t planning to deploy a sales force for its initial genetic mutations and will instead focus on “intensive efforts” around disease education and making its test more available, Meeker said. When doctors are confronted with a patient with abnormal hunger, they should be “thinking deep” about the cause, he added.

Spotlight

Cloud computing represents an IT service model which helps in the delivery of computing services, both hardware and software, to customers, on demand, over a network, which utilizes the internet. Thus, users only incur operating expenses associated with the time and length of usage, and are not required to make any initial capital expenditures.

Spotlight

Cloud computing represents an IT service model which helps in the delivery of computing services, both hardware and software, to customers, on demand, over a network, which utilizes the internet. Thus, users only incur operating expenses associated with the time and length of usage, and are not required to make any initial capital expenditures.

Related News

PHARMA TECH

Zerion Pharma and Hovione extend partnership to cover use of the Dispersome® technology platform in nutraceuticals

Zerion Pharma and Hovione | August 24, 2022

Hovione and Zerion Pharma announced an extension of their collaboration on Zerion's Dispersome® technology into the nutraceutical/dietary supplements field. Many dietary supplements suffer from low solubility. This results in poor bioavailability and consequently limits the physiological effect of the supplement. To overcome these limitations, the two companies will collaborate and apply the solubility enhancing Dispersome® technology for the development and commercialization of certain nutraceutical products. The first product candidate selected for joint development is an antioxidant with multiple health benefits and known for its extremely low solubility and bioavailability. By applying the Dispersome® technology, Zerion has been able to demonstrate significant solubility improvements of this antioxidant. Under their collaboration, Hovione and Zerion will now upscale and develop commercial formulations of the antioxidant using the Dispersome® platform and make these products available for distribution by partners globally. Under the terms of the collaboration agreement, the two companies will share income from the commercialization of these products according to their respective contributions. In addition to the joint development projects, Zerion has granted Hovione an exclusive license to exploit the Dispersome® technology for other nutraceuticals/dietary supplements. In return, Hovione will pay Zerion license fees and royalties on sales of the licensed products. "The low oral bioavailability of some of the health-promoting nutraceutical compounds is a well-known challenge. The problem is compounded by the fact that some of the solutions used in pharma cannot be used in foods. Hovione is thrilled to be Zerion´s exclusive partner for the application of Dispersome® to the fields of nutraceuticals and dietary supplements. The Dispersome® platform and its enabling ingredient – beta-lactoglobulin or BLG – afford formulators new options which address unmet needs of the industry." Jean-Luc Herbeaux, CEO of Hovione "I am extremely pleased with this extension of our collaboration", says Ole Wiborg, CEO of Zerion and continues: "The Dispersome® technology is actually very well suited for use in dietary supplements because it employs BLG as its solubility enabling component. BLG is a sustainable natural material and in itself a beneficial nutritional product that we source in high quality from Arla Food Ingredients. Since we as a company only have limited resources to exploit these promising applications of the Dispersome® technology in the nutraceutical field, the collaboration with Hovione is a win/win situation." In February 2022, Zerion and Hovione announced a strategic partnership aimed at commercializing the Dispersome® technology within the drug development field. Under this partnership, Hovione and Zerion are offering pharma and biotech companies worldwide access to an innovative drug delivery platform combined with an unparalleled experience in formulation development, scale up and GMP manufacturing. This unique combination provides customers in the pharma industry with a line of sight over the entire drug development life cycle from the preclinical phase to commercial drug product. About Hovione Hovione is an international company with over 60 years of experience as a Contract Development and Manufacturing Organization (CDMO) with a fully integrated offering of services for drug substance, drug product intermediate and drug product. The company has four FDA inspected sites in the USA, Portugal, Ireland and China and development laboratories in Lisbon, Portugal and New Jersey, USA. Hovione provides pharmaceutical customers services for the development and compliant manufacture of innovative drugs, including highly potent compounds, and customized product solution across the entire drug life cycle. In the inhalation area, Hovione offers a complete range of services, from API, formulation development and devices. Hovione´s culture is based on innovation, quality and delivery. Hovione was the first Chemical/ Pharmaceutical Company to become a Certified B Corp, is a member of Rx-360, EFCG and participates actively in industry quality improvement initiatives to lead new global industry standards. About Zerion Pharma A/S Zerion develops proprietary drug formulations and offers the Dispersome® technology to established pharma companies as a means to solve their most challenging drug solubility problems. By applying the Dispersome® technology, the solubility of poorly soluble, oral drugs is greatly enhanced, which improves their bioavailability and therapeutic outcomes for the patients. Zerion was established in 2019 as a spinout from the University of Copenhagen based on almost a decade of research.

Read More

PHARMA TECH

Armata Pharmaceuticals Announces Clearance of IND for Prosthetic Joint Infections

Armata Pharmaceuticals, Inc | August 02, 2022

Armata Pharmaceuticals, Inc. a biotechnology company focused on pathogen-specific bacteriophage therapeutics for antibiotic-resistant and difficult-to-treat bacterial infections, today announced that the U.S. Food and Drug Administration has cleared Armata's Investigational New Drug application for AP-SA02 in prosthetic joint infection. The company is initiating start-up activities for the Phase 1b/2a trial that will explore the safety, tolerability, and pharmacokinetics of intravenous and intra-articular doses of AP-SA02 as an adjunct to standard of care antibiotics in subjects with PJI. "Prosthetic joint infection is one of the most serious complications of prosthetic joint implantation. Staphylococcus aureus is a dominant pathogen that drives the need for surgical intervention and prolonged courses of antibiotic therapy. The ability of S. aureus to create biofilms that are refractory to standard of care antibiotics highlights the need for a novel therapy. Phage therapy may fill this need for PJI patients as phage have demonstrated the ability to disrupt biofilm and work synergistically with antibiotics." Dr. Mina Pastagia, Armata's Senior Vice President of Clinical Development "Armata is excited to explore AP-PA02 in patients who suffer from complicated joint infections that don't respond adequately to standard of care antibiotics. PJI is a logical extension of our diSArm trial which is targeting complicated S. aureus bacteremia," said Brian Varnum, Chief Executive Officer of Armata. "With this IND approval, Armata now has four active clinical programs. Today marks an important development milestone for Armata and its investors who set a course three years ago to transform phage therapy from a promising new technology to one that is explored in rigorous clinical settings." In addition to evaluating AP-SA02 in PJI and bacteremia due to S. aureus, Armata has advanced AP-PA02 into the Phase 2a component of the SWARM-P.a. study which targets chronic Pseudomonas aeruginosa infections in people with cystic fibrosis (CF). In February, the company gained IND clearance for AP-PA02 in a second indication, non-cystic fibrosis bronchiectasis targeting patients with chronic P. aeruginosa infections in a Phase 2 trial. About Armata Pharmaceuticals, Inc. Armata is a clinical-stage biotechnology company focused on the development of precisely targeted bacteriophage therapeutics for the treatment of antibiotic-resistant and difficult-to-treat bacterial infections using its proprietary bacteriophage-based technology. Armata is developing and advancing a broad pipeline of natural and synthetic phage candidates, including clinical candidates for Pseudomonas aeruginosa, Staphylococcus aureus, and other pathogens. In addition, in collaboration with Merck, known as MSD outside of the United States and Canada, Armata is developing proprietary synthetic phage candidates to target an undisclosed infectious disease agent. Armata is committed to advancing phage with drug development expertise that spans bench to clinic including in-house phage specific GMP manufacturing.

Read More

PHARMA TECH

Cadence Expands into Molecular Simulation with Acquisition of OpenEye Scientific, a Pioneering Leader in Computational Molecular Design

Cadence | July 26, 2022

Cadence Design Systems, Inc. announced that it has entered into a definitive agreement to acquire privately held OpenEye Scientific Software, Inc., a leading provider of computational molecular modeling and simulation software being widely and increasingly used by pharmaceutical and biotechnology companies for drug discovery. The addition of OpenEye’s technologies and experienced team with its deep scientific expertise accelerates Cadence’s Intelligent System Design™ strategy and expands its total addressable market bringing Cadence’s computational software expertise to apply proven algorithmic, simulation and solver advances to life sciences. Technological advancements, coupled with the rising demand for new drugs across a range of diseases, are driving increased demand for computational drug design. The growing utilization of computational techniques in drug discovery for pharmacology, chemical synthesis and drug screening to study the 3D structure of molecules is a major factor fostering the growth of the global biosimulation market, which has an estimated TAM of $2B and predicted to have a compound annual growth rate (CAGR) of approximately 15% over the next five years. Biosimulations are a critical tool for pharmaceutical research as they provide atomic-level insight into molecular interactions, and there’s a growing demand for high-performance simulation of larger biological systems over longer time scales. Cadence continues executing its Intelligent System Design strategy and has successfully leveraged its computational software expertise to expand into the system analysis space to address the electromagnetics, thermal and computational fluid dynamics (CFD) challenges in the high-tech electronics, aerospace and defense, and automotive sectors. Cadence is now extending that computational software core competency to molecular modeling and simulation that is targeted to life sciences. OpenEye, an industry leader in computational molecular design, has pioneered physics-based approaches and the cloud-native Orion® software platform to accelerate advances in human health. The acquisition allows pharmaceutical and biotechnology companies to benefit from more robust drug discovery solutions that combine OpenEye’s innovative molecular modeling and simulation software solutions for drug discovery with Cadence’s algorithmic and solver expertise, efficient large data management infrastructure, and leading AI/ML and cloud solutions. OpenEye’s products are used by 19 of the top 20 pharmaceutical companies globally—including Pfizer and AstraZeneca—as well as numerous biotechnology companies and academic institutions. Orion, OpenEye’s market-leading cloud-native software-as-a service (SaaS) platform, is growing rapidly as pharmaceutical and biotechnology users embrace its flexibility and scalability to perform complex molecular calculations on a large scale. “Drug discovery is an increasingly complex process that requires significant investment in research and development. The pending acquisition of OpenEye, with its scientifically tested methodologies and expertise, accelerates Cadence’s Intelligent System Design strategy with our entry into a new system domain of life sciences. Cadence’s deep computational software expertise drives further innovation in algorithms that enhance the reliability, efficiency and speed of molecular simulations. We look forward to welcoming such an accomplished team and are delighted to accelerate innovation and improve research and development productivity in the pharmaceutical and biotechnology industry.” Dr. Anirudh Devgan, president and CEO of Cadence “We founded OpenEye to stretch the boundaries of what’s possible in computational drug discovery,” said Dr. Anthony Nicholls, CEO of OpenEye Scientific. “Our 3D search technology, programming toolkits, and more recently, our cloud platform, Orion, have led the industry for more than 25 years, and this opportunity to join with Cadence to pursue even greater scale and accuracy is simply amazing. We foresee a time when pharmaceutical design, no matter the modality, is predominantly computational. The realization of that goal with our skills and Cadence’s resources and expertise is going to have a profound impact on human health.” Under the terms of the definitive agreement, Cadence will pay approximately $500 million in cash. The acquisition is expected to contribute immaterial revenue this year and about $40 million in fiscal 2023. It is expected to close in the third quarter of 2022, subject to completion of Hart-Scott-Rodino regulatory review and customary closing conditions. About Cadence Cadence is a pivotal leader in electronic systems design, building upon more than 30 years of computational software expertise. The company applies its underlying Intelligent System Design strategy to deliver software, hardware and IP that turn design concepts into reality. Cadence customers are the world’s most innovative companies, delivering extraordinary products from chips to boards to complete systems for the most dynamic market applications, including hyperscale computing, 5G communications, automotive, mobile, aerospace, consumer, industrial and healthcare. For eight years in a row, Fortune magazine has named Cadence one of the 100 Best Companies to Work For. About OpenEye Scientific OpenEye Scientific is an industry leader in computational molecular design through rapid, robust and scalable software, toolkits, technology and design services, and Orion®, the only cloud-native fully integrated software-as-a-service molecular modeling platform that runs on Amazon Web Services. Combining unlimited computation and storage with powerful tools for data sharing, visualization and analysis in an open development platform, Orion offers unprecedented capabilities for the advancement of pharmaceuticals, biologics, agrochemicals, and flavors and fragrances. Founded in 1997, OpenEye Scientific is a privately held company headquartered in Santa Fe, N.M., with offices in Boston, Mass.; Cologne, Germany; and Tokyo, Japan.

Read More