Ultra-Rare to Rare: Two Execs Talk About Alexion's Shift

biospace | May 09, 2019

Alexion Pharmaceuticals is a company focused on ultra-rare diseases, although it is working on a shift into rare diseases. This may sound like something of a minor shift, but it represents developing drugs for more diseases with larger patient populations. The company’s success has been built primarily on its Soliris (eculizumab), a complement inhibitor. The drug has been approved for paroxysmal nocturnal hemoglobinuria (PNH), atypical Hemolytic Uremic Syndrome (aHUS) and for generalized Myasthenia Gravis (gMG). Soliris, as well as the company’s next-generation complement inhibitor, Ultomiris, underline the company’s expertise in complement-related diseases. The complement system is part of the immune system made up of about 30 different proteins. As such it plays a role in numerous diseases at some level, and alone in several diseases, including PNH, aHUS and others.

Spotlight

Sanofi – Sleeping Sickness - Across many parts of Africa, some 65 million people are at risk of contracting sleeping sickness. This video shows the history of the development of a treatment for this disease.

Spotlight

Sanofi – Sleeping Sickness - Across many parts of Africa, some 65 million people are at risk of contracting sleeping sickness. This video shows the history of the development of a treatment for this disease.

Related News

PHARMA TECH

Ranok Therapeutics Announces Agreement with Pfizer on Targeted Protein Degradation

Ranok Therapeutics | November 30, 2021

Ranok Therapeutics Co. Ltd., an emerging biopharmaceutical company focused on developing breakthrough therapies for cancer and other serious diseases, announced an agreement with Pfizer Inc. to apply and evaluate Ranok’s CHAMP™ platform technology on an undisclosed cancer target. Terms of the agreement were not disclosed. Ranok has developed a proprietary and innovative targeted protein degradation (TPD) platform technology, CHAMP™ (Chaperone-mediated Protein Degradation), which leverages Ranok’s founders’ expertise in protein homeostasis to take advantage of the cellular chaperone network, potentially improving drug safety and efficacy due to selective targeting of disease tissues. “We are very pleased to work with Pfizer to explore the application of CHAMP™ to an emerging cancer drug target, This represents our first pharmaceutical company research relationship and is an important step forward in establishing CHAMP™ as a preferred technology in the rapidly-growing field of targeted protein degradation. We look forward to uncovering new benefits of CHAMP™ through this research.” Kevin P. Foley, Ph.D., Co-founder and Chief Scientific Officer of Ranok Therapeutics This evaluation agreement builds upon Ranok’s recent momentum, including its $40 million Series B financing in August 2021. About Ranok Therapeutics Ranok is a privately held, emerging biopharmaceutical company that is pioneering its proprietary CHAMP™ platform technology, an innovative approach to targeted protein degradation (TPD) for the discovery and development of novel therapeutics. Ranok was founded by a leadership team composed of industry veterans from both Chinese and American biotech and pharmaceutical companies and is based in two of the world’s leading centers of innovation and medical research: Hangzhou China and Greater Boston, Massachusetts. By bringing together cutting-edge medical research from both China and the United States, Ranok’s goal is to create transformative medicines to benefit patients suffering from cancer and other serious diseases around the world. For more information, please visit the company website at www.ranoktherapeutics.com or follow us at LinkedIn and on Twitter @RanokTx. About Ranok’s CHAMP™ platform Chaperone-mediated Protein Degradation/Degrader technology is based on our founders’ extensive backgrounds in protein homeostasis research. This technology takes advantage of the cellular chaperone network, distinguishing it from other targeted protein degradation (TPD) approaches. CHAMP™ has a number of unique advantages, including improved safety due to the selective targeting of tumors. Our proprietary R&D engine combines deep biological insight and chemistry expertise to iteratively discover and test new leads, rapidly identifying and advancing optimal development candidates for a variety of important disease targets.

Read More

BUSINESS INSIGHTS

Health Canada Approves Ipsen’s Sohonos™ (palovarotene capsules) as the First Approved Treatment for Fibrodysplasia Ossificans Progressiva

Ipsen | January 24, 2022

Ipsen announced the Health Canada approval of Sohonos, an oral selective retinoic-acid receptor gamma agonist indicated to reduce the formation of heterotopic ossification in adults and children aged 8 years and above for females and 10 years and above for males with fibrodysplasia ossificans progressiva.1 Sohonos is approved for the treatment of patients with FOP for both chronic use, and for flare-ups, in these patient populations. This decision marks the first approval for Sohonos worldwide. “FOP is a progressive and debilitating condition which has such a profound impact on patients, and their families. Until today, there was no approved medicine, and we are proud to bring this important new medicine to the FOP community.” Dr. Howard Mayer, Executive Vice President and Head of Research and Development, Ipsen FOP is characterized by new bone formation outside of the normal skeletal system, like in soft connective tissues, a process known as heterotopic ossification,2 which can be preceded by painful soft tissue swelling or “flare-ups.”2 Flare-up episodes are common and are a substantial contributor to the formation of new bone, however bone can also form in the absence of a flare-up. Once formed, it is irreversible and leads to loss of mobility and shortened life expectancy.2 It is an ultra-rare genetic disorder with an estimated prevalence of 1.36 per million individuals; however, the number of confirmed cases varies by country.3,4. As part of the ongoing commitment to the FOP and rare disease community, Ipsen plans to file in the US in H1 2022 and is in discussions with other regulatory authorities around the world. About the MOVE clinical program This approval is based on data from the ongoing MOVE trial, the first global multi-center Phase III trial in FOP. MOVE is an open-label, single-arm trial, evaluating the efficacy and safety of a chronic/flare-up dosing regimen of palovarotene in decreasing new annualized HO volume in patients with FOP. About Sohonos Sohonos is an oral, selective RARγ agonist developed as a treatment for people living with the debilitating ultra-rare genetic disorder, FOP. The treatment was acquired by Ipsen through the acquisition of Clementia Pharmaceuticals in April 2019. It is a member of the retinoid class of drugs that is associated with birth defects in humans. Sohonos must not be used by patients who are, or intend to become, pregnant due to the risk of teratogenicity. To minimize fetal exposure, Sohonos is to be administered only if all conditions for pregnancy prevention are met. Sohonos has been shown to cause premature physeal closure in growing children with FOP; periodic monitoring is recommended. About Ipsen Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience; it also has a well-established Consumer Healthcare business. With total sales of over €2.5bn in FY 2020, Ipsen sells more than 20 medicines in over 115 countries, with a direct commercial presence in more than 30 countries. The Company’s research and development efforts are focused on its innovative and differentiated technological platforms located in the heart of leading biotechnological and life-science hubs: Paris-Saclay, France; Oxford, U.K.; Cambridge, U.S.; Shanghai, China. Ipsen has around 5,700 colleagues worldwide and is listed in Paris and in the U.S. through a Sponsored Level I American Depositary Receipt program.

Read More

RESEARCH

Moderna and Medison Pharma Collaborate to Commercialize Moderna's COVID-19 Vaccine in Central Eastern Europe and Israel

Moderna | June 08, 2021

Moderna, Inc., a biotechnology company that pioneered messenger RNA (mRNA) therapeutics and vaccines, and Medison Pharma, a leading commercial partner for highly innovative therapies in international markets, announced today a new agreement to commercialize the Moderna COVID-19 Vaccine in Central Eastern Europe and Israel. Poland, Czech Republic, Romania, Hungary, Bulgaria, Slovenia, Slovakia, Croatia, Estonia, Latvia, Lithuania, Serbia, Ukraine, Moldova, Albania, Bosnia, and Herzegovina, Kosovo, North Macedonia, Montenegro, and Israel are among the nations included by the pact. "We are excited to work with Moderna in 20 markets, covering over 175 million people across the whole Central Eastern Europe region and in Israel," stated Meir Jakobsohn, Medison Pharma's Founder and CEO. "With Moderna's ground-breaking mRNA vaccine and Medison's international commercialization platform for highly innovative treatments, our collaboration is a perfect fit." "We value this new collaboration with Medison Pharma to ensure the successful launch of our mRNA COVID-19 vaccine," stated Corinne Le Goff, Pharm.D., M.B.A., Chief Commercial Officer of Moderna. "By delivering our vaccines in collaboration with our partners, we stay consistent in our commitment to fighting the pandemic." About Medison Pharma Medison is a global pharmaceutical company that focuses on commercializing highly innovative therapies in international markets. Medison offers a full range of integrated services for companies looking to establish a presence in international markets, drawing on a 25-year track record and collaborations with major biotech companies. Medison also has a corporate venture arm with a dedicated research and evaluation team with significant scientific and commercial expertise. About Moderna Moderna has evolved from a science research-stage company advancing programs in the field of messenger RNA (mRNA) to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across six modalities, a broad intellectual property portfolio in areas such as mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant. Moderna maintains alliances with a diverse range of domestic & global government and commercial collaborators, allowing for the pursuit of both ground-breaking science and rapid manufacturing scale.

Read More