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Developing drugs to treat rare diseases is fraught with challenges; these range from trying to recruit from tiny patient populations to fill much-need clinical trials to the complex reimbursement landscape for these innovative, and often bespoke, therapies. GlobalData’s Pharmaceutical Technology writer Allie Nawrat looks at three case studies of companies on the verge of having treatments for largely ignored rare diseases approved. Restoring DNA repair functionality to cells: Rocket’s Fanconi gene therapy Nawrat Says: “Fanconi anaemia (FA) is a rare paediatric inherited disease caused by a mutation in the FANC genes. Patients with Fanconi experience bone marrow failure as they are unable to create new blood cells. “Rocket wants to change this situation with its lentiviral vector gene the…
19th February 2020: Cegedim UK announces the launch of a new Mobile App for Healthcare Professionals. The app showcases Cegedim’s powerful clinical decision support web-services which everyday enable over 5,000 pharmacies to dispense and over 4,000 GP’s to safely select and prescribe medicines for patients. The app brings together technology and data to demonstrate the novel capabilities of Cegedim’s solutions to support clinicians, solutions providers and healthcare providers to provide safe and effective care in a scalable and cost effective package. The new online application is aimed at GPs, pharmacists and other healthcare professionals and provides access to up to date, unbiased and reliable medical information, for optimum clinical decision-making.  The App references Cegedim’s drug…
Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), today announced the formation of Chiesi Global Rare Diseases, a new business unit that will harness the full resources of the Chiesi Group to advance research and new product development for rare and ultra-rare diseases. The unit will be headquartered in Boston, Massachusetts and have an initial focus on research and product development in lysosomal storage disorders, rare haematology and ophthalmology disorders. “Chiesi has a long history of success in discovering, developing and commercialising innovative therapies to address unmet needs for people living with rare diseases. With the Chiesi Global Rare Diseases unit, we are taking this to an entirely new level – rededicating and strengthening o…
The Wuhan coronavirus outbreak shows no signs of slowing down, and developments in the unfolding story are so rapid that it can be difficult keep track of them. GlobalData’s Pharmaceutical Technology team have produced a free-to-access dashboard to keep industry, media, and the public, up-to-date with the latest developments in the coronavirus outbreak and a range of tools to provide key information. The dashboard contains: Breaking news and regional reports Maps and trackers of confirmed cases, global spread, deaths and recoveries Timeline of events from the first cases to the latest news Social media tracking analysing trending keywords, hashtags, mentioned companies and countries An FAQ resource answering common questions Click on the link below to visit the web page. https://www.phar…
Manchester, UK, 30th January 2020 – Chiesi, an international research-focussed pharmaceutical group (Chiesi Group), has been awarded the exclusive Top Employers UK and the Top Employers Europe for the eighth consecutive year running following an extensive review process by the Top Employers Institute. The Top Employers Institute recognises excellence in people and working practices, through analysing data and information relating to the Human Resource management policies of global companies. To achieve certification, a company is assessed based on a thorough process of internal research, to demonstrate it complies with the most stringent quality standards in a range of areas, including training and development, strategies aimed at nurturing talent and company culture. Tom Delahoyde, Managi…
Both Edwards Lifesciences and Teva won four Golds and one Silver each, making them joint top of the pharma charts. Roche won two Golds and two Silvers and Reckitt Benckiser (RB) two Golds. Other Gold winners on the client side included ALK, Heart UK, Immunocore, Janssen, Mundipharma, Novo Nordisk and Santen.    The event is the largest in the industry awards calendar and attracts around 900 people from across pharma companies and agencies to the largest ballroom in Park Lane. The ceremony was skilfully hosted by Claudia Winkleman in combination with comedian Zoe Lyons and everyone’s favourite voice, Alan Dedicoat, the voice of Strictly.  Although the Gold Awards were dominated by Havas Lynx Group and VCCP, there were 21 different agencies and 30 different pharmaceutical or healthcare organ…
From an increase in sales of interleukin inhibitors to the prevalence of biosimilars, this decade has been an eventful one in the immunology industry. Patrick Aiyes, Senior Immunology Analyst at leading data and analytics company GlobalData, observes the key trends that has shaped the immunology market in the last decade: Key immunology drugs “The immunology market has seen a continuous increase in the sales of interleukin inhibitors and a decrease in the sales of TNF inhibitors - primarily because of the entry of biosimilars into the market and the increased safety profile associated with interleukin inhibitors. Abbvie’s Humira will end the decade as the highest grossing drug of 2019, generating sales of approximately $19bn globally, while Janssen’s Stelara will be the highest-grossing i…
Fasenra being evaluated in eight eosinophil-driven diseases beyond severe asthma AstraZeneca today announced three new trials for Fasenra (benralizumab) in skin diseases, adding to five trials already underway for the medicine in eosinophil-driven diseases (EDDs) beyond severe asthma. In EDD, immune system dysfunction causes eosinophil recruitment and activation leading to chronic local and/or systemic inflammation.1 The three new trials for Fasenra in skin diseases include two Phase II trials to assess the potential of the medicine in atopic dermatitis (AD) and chronic spontaneous urticaria (CSU); and a Phase III trial in bullous pemphigoid (BP). Five additional Phase III clinical trials are underway to investigate the potential of Fasenra in: Disease Trial name Estimated data readout …
Switzerland, January 15th 2020 – Oviva, the leading digital provider of Type 2 diabetes treatment in Europe, has raised $21m in Series B funding. MTIP led the round, joined by Earlybird as new investors. Existing investors AlbionVC, F-Prime Capital, Eight Roads Ventures and Partech all participated. The new capital will be used to further develop Oviva’s technology and expand in Europe to serve the millions of patients not accessing treatment today. It brings the total amount raised by Oviva to date to $34m. Oviva offers an evidence-based digital solution to stop the progression of and reverse Type 2 diabetes and obesity-related conditions. Patients receive tailored nutrition advice and personalised coaching via their phone. Oviva’s technology-supported treatment has consistently demonstra…
BIOGEN TO ACQUIRE NOVEL CLINICAL STAGE ASSET WITH APPLICATION IN ALZHEIMER’S DISEASE AND PARKINSON’S DISEASE FROM PFIZER INC PF-05251749 is a CNS-penetrant regulator of circadian rhythm with potential to address behavioral and neurological symptoms across various psychiatric and neurological diseases  Biogen to pay Pfizer $75 million upfront plus potential milestones of up to $635 million, and royalties  PF-05251749 complements the Company’s pipeline of potential disease-modifying therapies for Alzheimer’s and Parkinson’s diseases CAMBRIDGE, Mass., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced an agreement to acquire from Pfizer Inc. (NYSE: PFE) PF-05251749, a novel CNS-penetrant small molecule inhibitor of casein kinase 1 (CK1), for the potential treatment…
MONACO, PRINCIPALITY OF MONACO, AND NEW YORK, NY, USA, January 14, 2020: Lyfebulb, a patient empowerment platform that connects patients with industry to support user-driven innovation toward solutions in chronic disease, and Helsinn, a Swiss pharmaceutical group focused on building quality cancer care, announce the 11 Patient Entrepreneurs chosen as finalists for the Lyfebulb-Helsinn Cancer Innovation Summit & Award to be held on January 30, 2020. The following inspirational finalists represent companies that have been founded by cancer patients, cancer survivors or those with loved ones affected by cancer: Alexander Börve, PhD of First Derm Jakub Chudik of ConquerX Russell LaMontagne of Boston Immune Technologies and Therapeutics Robert Manning of CureMatch Gilles Pagès, PhD of InfA…
Ashfield Pharmacovigilance Inc. becomes part of Ergomed’s PrimeVigilance division, bolstering its market-leading position as a global independent specialist pharmacovigilance provider and establishing a platform for the broader Ergomed services business in the US Combined business expands PrimeVigilance’s geographic coverage in the strategically important US market and strengthens global service offering Acquisition adds over 40 new clients to PrimeVigilance and a strong order book of contracted future revenues of $9.8 million Ashfield Pharmacovigilance Inc. reported $11.6 million revenue and $0.9 million adjusted EBITDA in the year to 30 September 2019 and is expected to be immediately accretive to earnings   London, UK – 13 January 2020: Ergomed plc (LSE: ERGO) ("Ergomed" or the "Compa…
Almirall has an option right to acquire Bioniz Therapeutics, Inc. which will give full global rights to BNZ-1 BNZ-1 is a multicytokine inhibitor that selectively blocks the activity of three cytokines of the same family: IL-2, IL-9 and IL1-15, currently in phase 1/2 development for refractory Cutaneous T-cell Lymphoma If Almirall exercises the option, it will also enter into a broader research agreement with Bioniz NewCo, using its multiple cytokine inhibitor platform with the objective to deliver at least 3 IND-approved candidates Almirall, S.A. (ALM) has announced today the execution of an option agreement to acquire Bioniz Therapeutics, Inc. a clinical stage biopharmaceutical company based in Irvine, California, which develops first-in-class peptide treatments that selectively inhibit…
GlaxoSmithKline plc (LSE/NYSE: GSK) has announced the completion of the divestment of travel vaccines Rabipur (tradename Rabavert in the US) for the prevention of rabies, and Encepur for the prevention of tick-borne encephalitis, to Bavarian Nordic. Consistent with the terms of the agreement first announced on October 21st 2019, GSK has received an upfront payment of EUR308 million (£263m), and in due course will receive further milestone payments for a total consideration of up to EUR955 million. The decision to divest these brands supports GSK’s strategic intent to increase focus and reinvest in growth assets, innovation and a simplified supply chain in its vaccines business. About Rabipur (Tradename Rabavert in US and Canada)Rabipur is a well-established life-saving vaccine (rab…
02-Jan-2020
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PARIS – December 23, 2019 – Sanofi announced today that it intends to commence a tender offer (the “Offer”) today to acquire all of the outstanding shares of common stock of Synthorx, Inc. (“Synthorx”) for $68 per share in cash, without interest thereon and net of any applicable withholding taxes. The Offer is being made pursuant to the Agreement and Plan of Merger, dated as of December 7, 2019 (as it may be amended from time to time, the “Merger Agreement”), by and among Synthorx, Sanofi and Thunder Acquisition Corp., a Delaware corporation and an indirect, wholly-owned subsidiary of Sanofi (“Purchaser”). The Offer is scheduled to expire one minute past 11:59 p.m., Eastern Time, on Wednesday, January 22, 2020, unless the Offer is extended in accordance with the Merger Agreement and the…
Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, today announces Syncona Ltd (“Syncona) has made an $80.0 million (£61.1 million)[1] commitment in a Series C financing and has funded the first tranche of this commitment, amounting to $40.0 million (£30.6 million)[2]. This financing augments the previous £88.4 million Series B investment and the £34.8 million Series A investment previously made into Freeline by the founding investors, led by Syncona. The first $40 million tranche will enable Freeline to expand its team, continue to develop its robust manufacturing platform, including commercial supply capability, generate further data in its clinical programmes for Haemophilia B and Fabry Disease, and continue to progress its pip…
PARIS and San Diego, California – December 9, 2019 – Sanofi and Synthorx, Inc. (NASDAQ: THOR), a clinical-stage biotechnology company focused on prolonging and improving the lives of people suffering from cancer and autoimmune disorders, entered into a definitive agreement under which Sanofi will acquire all of the outstanding shares of Synthorx for $68 per share in cash, which represents an aggregate equity value of approximately $2.5 billion (on a fully diluted basis). The transaction was unanimously approved by both the Sanofi and Synthorx Boards of Directors. “This acquisition fits perfectly with our strategy to build a portfolio of high-quality assets and to lead with innovation, as you will hear at our Capital Markets Day tomorrow, December 10. Additionally, it is aligned with our go…
Craigavon, N.I., 6 December 2019 –  Almac Group is delighted to announce Almac has won the “Innovation in Sponsorship” award during this year’s Irish Sponsorship Awards ceremony in Dublin. This award, given annually, recognizes “a distinct new method or unique approach to a sponsorship activation, partnership or initiative” and we received the award in recognition of our unique and innovative £1 million sponsorship agreement with W5, the interactive discovery center in Belfast. As announced previously, this partnership, a first of its kind in Ireland, has resulted in a comprehensive STEM learning program based at W5 that aims to improve access to STEM education and promote exciting career possibilities available through pursuing STEM subjects. As a business employing over 5,600 individuals…