Saniona Receives U.S. FDA Orphan Drug Designation for Tesomet in Hypothalamic Obesity
July 26, 2021
Tesomet is the first and only investigational treatment for hypothalamic obesity to receive orphan drug designation
Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Tesomet for the treatment of hypothalamic obesity (HO). Tesomet is the first and only investigational treatment for HO to receive orphan drug designation. Saniona is preparing to initiate two Phase 2b studiesof Tesomet in the second half of this year, one in HO and the other in Prader-Willi syndrome (PWS), for which Tesomet has already received orphan drug status.
“There are currently no FDA-approved medicines for hypothalamic obesity. Despite the devastating weight gain and hunger this rare disease can cause, there has been relatively little drug development specifically for HO. Saniona is proud to be pioneering a regulatory path forward for people living with HO, and we are thrilled to have received the first-ever FDA orphan drug designation in HO. We look forward to initiating our Phase 2b trial of Tesomet for HO in the second half of this year,” said Rudolf Baumgartner, M.D., Chief Medical Officer and Head of Clinical Development at Saniona.
Amy Wood, Executive Director of the Raymond A. Wood Foundation and parent of a child living with hypothalamic obesity, commented, “The recognition of the first orphan drug designation in hypothalamic obesity is a critical milestone for the HO community. HO places a tremendous burden on caregivers and families; it causes severe weight gain and constant hunger no matter how much a person eats, forcing us to lock up food and avoid social situations where food is served. We are incredibly grateful that both the FDA and Saniona recognize the seriousness of this disorder, and we hope this orphan drug designation is the first step towards having an innovative treatment.”
Orphan drug designation is a special status granted by the FDA to medicines and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. The number of people living with HO is estimated to be between 10,000 and 25,000 in the U.S. and between 16,000 and 40,000 in Europe. Receiving orphan designation qualifies Saniona for certain development benefits, including tax credits, elimination of certain FDA license application fees, and seven years of market exclusivity in the U.S. following approval.
Saniona previously evaluated Tesomet in a 24-week, double-blind, randomized, placebo-controlled initial Phase 2 trial in HO. Adults receiving Tesomet demonstrated statistically significant reductions in body weight and improvements in waist circumference and glycemic control. These improvements were maintained during an additional 24-week open-label extension. Tesomet was reported to be generally well-tolerated, and most adverse events were mild. The most common adverse events included sleep disorders, dizziness, dry mouth and headache. No clinically significant differences in heart rate or blood pressure were observed between the Tesomet-treated patients and the placebo-treated patients.
In preparation for the initiation of the Phase 2b study of Tesomet in HO in the second half of 2021, Saniona has selected the clinical research organization (CRO) that will support the clinical trial and is in the process of assessing and selecting clinical trial sites in the U.S. and globally. Saniona has also selected the contract manufacturer to produce Tesomet for Phase 2b and Phase 3 clinical trials. Additionally, Saniona has initiated multiple partnerships with the HO advocacy community to incorporate caregiver and patient feedback into the clinical trial process and to provide the community with education on clinical trials.
Saniona is also evaluating Tesomet for the treatment of Prader-Willi syndrome (PWS) and plans to begin a Phase 2b trial in this indication in the second half of this year. The FDA granted orphan drug designation to Tesomet for the treatment of PWS in March 2021.
For more information, please contact
Trista Morrison, Chief Communications Officer, Saniona. Office: + 1 (781) 810-9227. Email: email@example.com
This information is such information as Saniona AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 8:00 CEST on 26July 2021.
Saniona is a biopharmaceutical company focused on discovering, developing, and delivering innovative treatments for rare disease patients around the world. The company’s lead product candidate, Tesomet, is in mid-stage clinical trials for hypothalamic obesity and Prader-Willi syndrome, severe rare disorders characterized by uncontrollable hunger and intractable weight gain. Saniona’s robust drug discovery engine has generated a library now consisting of more than 20,000 proprietary modulators of ion channels, a significantly untapped drug class that is scientifically validated. Lead candidate SAN711 is in a Phase 1 clinical trial and may be applicable in the treatment of rare neuropathic disorders, and SAN903 for rare inflammatory and fibrotic disorders is advancing through preclinical development. Led by an experienced scientific and operational team, Saniona has an established research organization in Copenhagen, Denmark and a corporate office in the Boston, Massachusetts area, U.S. The company’s shares are listed on Nasdaq Stockholm Small Cap (OMX: SANION). Read more at www.saniona.com.
Tesomet is an investigational fixed-dose combination therapy of tesofensine (a triple monoamine reuptake inhibitor) and metoprolol (a beta-1 selective blocker). Saniona is advancing Tesomet for hypothalamic obesity and Prader-Willi syndrome, two severe rare disorders characterized by obesity and loss of appetite control. The programs are currently in clinical development. Saniona holds worldwide rights to Tesomet and is actively evaluating opportunities to advance this treatment globally.
About Hypothalamic Obesity (HO)
Hypothalamic obesity (HO) is a rare disorder caused by injury to a region of the brain known as the hypothalamus. This injury is most commonly sustained during surgery to remove a noncancerous tumor called a craniopharyngioma (CP). HO is characterized by rapid, excessive, and intractable weight gain that persists despite restricted food intake. Patients may have hyperphagia, an uncontrollable hunger, and may display abnormal food seeking behavior such as stealing food. Additional symptoms may include memory impairment, attention deficit, excessive daytime sleepiness and lethargy, issues with impulse control, and depression. HO patients are also at increased risk of developing obesity-related comorbid conditions such as Type 2 diabetes, non-alcoholic fatty liver disease, hypertension, stroke, and congestive heart failure. Ultimately, CP survivors with extensive hypothalamic injury report a 20-year mortality rate at least three times higher than CP survivors without extensive hypothalamic injury. There are no medications approved specifically for HO, and there is no cure for this disease. Many HO patients are treated with approaches used for general obesity such as surgery, medication and lifestyle counseling, but these are mostly ineffective. The number of patients with of HO is estimated to be between 10,000 and 25,000 in the U.S. and between 16,000 and 40,000 in Europe.
AttachmentTo view this piece of content from www.globenewswire.com, please give your consent at the top of this page.
To view this piece of content from ml-eu.globenewswire.com, please give your consent at the top of this page.
One Liberty Plaza - 165 Broadway
NY 10006 New York
GlobeNewswire is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.
Subscribe to releases from GlobeNewswire
Subscribe to all the latest releases from GlobeNewswire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from GlobeNewswire
Another important step for dLab and Dala Energi28.9.2021 09:00:00 CEST | Press release
We are happy to announce that our journey with Dala Energi takes yet another important step! The product dAnalyzer Lite from cleantech company Dlaboratory Sweden AB (dLab), are being integrated to one more station, Lindan. Dala Energi's business concept is to offer solutions in energy and digital communication that help people and companies live and develop in our countryside in an easy, efficient, and sustainable way and dLab is happy to support Dala Energi in realizing their mission. dLab’s dInsight Analytics Platform provides a decision support system including a broad set of features and services. One of these features is dAnalyzer, collecting grid data from its minimal hardware, completely independent of age and make, including high-resolution sensors. The data is analyzed and put into meaningful context through a web-based interface; thereby enabling proactive fault detection as well as proactive maintenance. In modern substations equipped with digital protection relays including
DNO Updates North Sea Drilling28.9.2021 08:27:36 CEST | Press release
Oslo, 28 September 2021 – DNO ASA, the Norwegian oil and gas operator, today reported an oil discovery on the Gomez prospect in its operated PL006C license offshore Norway. The exploration well encountered hydrocarbons in the primary target in the Våle Formation of Paleocene Age. The reservoir is a 23 meters thick, homogeneous sandstone of poor to moderate quality. A small amount of oil was recovered during logging. The oil/water contact was not encountered. Based on preliminary assessments, there is uncertainty whether the reservoir can be commercially produced and no estimate of recoverable volumes has been established at this stage. DNO holds a 65 percent interest in the PL006C license and together with its partner Aker BP (35 percent) will study the extensive data collected during the operation before deciding next steps. In a separate announcement, Equinor as operator of license PL159B (DNO 32 percent) has reported that the recently completed Black Vulture exploration well did not
Statkraft sells Andershaw Wind Farm and retains long-term management role28.9.2021 08:10:13 CEST | Press release
- Greencoat UK Wind PLC has purchased 100% of Statkraft’s ownership in Andershaw Wind Farm - Statkraft to retain long-term operations and maintenance as well as asset management - The two companies have also signed an extension of the existing PPA agreement (UK and Norway, 28 Sept 2021) Statkraft, Europe’s largest generator of renewable energy, has today announced the sale of Andershaw Wind Farm in Scotland, to Greencoat UK Wind PLC for a purchase price of £121m (including cash and working capital). Statkraft, who together with Catamount Energy*, began joint development of the project in 2006 and operation in 2017, will maintain day-to-day operation and maintenance, as well as asset management responsibilities until 2037. The company has also negotiated an extension of its existing long-term market access power purchase agreement. * Andershaw Wind Farm (36 MW installed capacity), south of Glasgow, produces enough energy to meet the annual needs of about 26,000 homes. Eivind Torblaa, Vi
Statkraft selger Andershaw Wind Farm i Skottland og fortsetter å drifte vindparken28.9.2021 08:10:13 CEST | Pressemelding
- Greencoat UK Wind PLC har kjøpt hele Andershaw Wind Farm fra Statkraft - Statkraft vil levere alle drifts- og vedlikeholdstjenester for vindparken til Greencoat UK Wind - Partene har også forlenget en eksisterende kraftkjøpsavtale (Oslo/London, 28. september 2021) Statkraft, Europas største produsent av fornybar energi, har solgt Andershaw Wind Farm i Skottland til Greencoat UK Wind PLC for 121 millioner pund, inkludert kontanter og arbeidskapital. Statkraft startet utviklingen av prosjektet i 2006 og vindparken ble satt i drift i 2017. I forbindelse med salget er det avtalt at Statkraft skal fortsette å drifte anlegget, samt levere alle vedlikeholds- og eieroppfølgingstjenester til Greencoat fram til 2037. En forlengelse av den eksisterende kraftkjøpsavtalen er også avtalt. Vindparken som ligger sør for Glasgow har elleve turbiner, en samlet installert effekt på 36 MW og produserer nok fornybar energi til å dekke forbruket i rundt 26.000 britiske husstander. Eivind Torblaa, Vice Pre
Teledyne to showcase comprehensive portfolio of industrial and scientific imaging technology at Vision 202128.9.2021 08:00:00 CEST | Press release
MUNICH, Germany, Sept. 28, 2021 (GLOBE NEWSWIRE) -- Teledyne, a part of Teledyne Technologies [NYSE: TDY], and a global leader in machine vision technology, will exhibit their newest technologies at Vision 2021 taking place October 5-7, 2021 in Stuttgart, Germany. Teledyne will display the world’s most comprehensive, vertically integrated portfolio of industrial and scientific imaging technology including the latest from newly acquired Teledyne FLIR. Visit the Teledyne booth and explore the unmatched capabilities and products from Teledyne’s DALSA, e2v, FLIR, and Lumenera business units. Your one source for unlimited vision will be on display at Stand 8 B10. Teledyne will participate in the Industrial Vision Days with keynote and technology presentations taking place Wednesday, October 6, 2021: At 9:20 AM join Teledyne DALSA’s Matthias Sonder delivering his presentation entitled “Clarity at High Speed” At 4:20 PM be sure to attend Teledyne e2v’s Sergio Morillas speaking about “Highly r
Ahold Delhaize share buyback update28.9.2021 08:00:00 CEST | Press release
Zaandam, the Netherlands, September 28, 2021 – Ahold Delhaize has repurchased 606,980 of Ahold Delhaize common shares in the period from September 20, 2021 up to and including September 24, 2021. The shares were repurchased at an average price of €28.55 per share for a total consideration of €17.3 million. These repurchases were made as part of the €1 billion share buyback program announced on November 4, 2020. The total number of shares repurchased under this program to date is 28,367,721 common shares for a total consideration of € 690.4 million. Download the share buyback transactions excel sheet for detailed individual transaction information from https://www.aholddelhaize.com/en/investors/share-buyback-programs/2021/ This press release is issued in connection with the disclosure and reporting obligation set out in Article 2(2) of the EU Regulation that contains technical standards for buyback programs.
ASM INTERNATIONAL HOSTS INVESTOR DAY, UPDATES Q3 2021 GUIDANCE28.9.2021 07:45:00 CEST | Press release
Almere, The Netherlands September 28, 2021, 7:45 a.m. CET ASM International N.V. (Euronext Amsterdam: ASM) today hosts its 2021 Investor Day. ASM today also announces that the order intake in Q3 2021 is expected to exceed previous guidance. ASM holds today its Investor Day. During this event ASM’s management will provide an update on the company’s strategy, how we are going to create value and drive growth through innovation Highlights include: Targeting revenue of €2.8-€3.4 billion by 2025 (2020-2025 CAGR of 16%-21%), gross margins of 46%-50% in 2021-2025, and operating margins of 26%-31% in 2021-2025;Targeting Net Zero emissions by 2035 and 100% renewable electricity by 2024;1Single-wafer ALD market expected to increase from $1.5 billion in 2020 to $3.1-3.7 billion by 2025;Further capacity expansion of our new manufacturing facility to be production ready by early 2023. “I’m very excited to share the strong prospects for our company during our first Investor Day,” said Benjamin Loh,