Ascendis Pharma A/S

If approved, eftansomatropin alfa will enter a market that already features a once-weekly option. Genexine’s once-weekly growth hormone has matched daily injections in a pivotal trial in China, teeing it and its partners I-Mab and Jumpcan up to file for approval next year and prepare to launch the medicine.  The phase 3 clinical trial compared eftansomatropin alfa, Genexine’s long-acting growth hormone, to Novo Nordisk’s daily injectable Norditropin in children with growth hormone deficiency. After 52 weeks, the mean annualized height velocity in the eftansomatropin alfa arm was 10.76 cm a year, compared to 10.28 cm a year in the control cohort. The result caused the trial to meet its primary endpoint. “We are pleased with the results from this registration trial as this demonstrates clear safety and efficacy in this potential best-in-class drug to treat children with growth hormone deficiency,” Genexine CEO Neil Warma said in a statement. “This is a significant milestone for Genexine as we demonstrate our ability to bring our novel products to market and the value of our proprietary long-acting hyFc platform.” Genexine granted Tasgen Biotech, a company subsequently bought by I-Mab, the rights to develop, manufacture and commercialize eftansomatropin alfa in China in 2015 as part of a multi-asset deal worth $13 million upfront plus up to $40 million in milestones tied specifically to the growth hormone. I-Mab partnered with Jumpcan to commercialize the drug in China in 2021. The collaborators plan to file for approval in China next year. If approved, eftansomatropin alfa will enter a market that already features a once-weekly option. GenSci won approval for its once-weekly Jintrolong in China after showing the treatment is noninferior to daily injections.  Jintrolong and eftansomatropin alfa are part of a wave of once-weekly growth hormone drugs including Ascendis Pharma’s Skytrofa and Pfizer’s Ngenla. The developers have achieved once-weekly dosing using a variety of approaches. Jintrolong is a pegylated growth hormone, whereas eftansomatropin alfa uses hyFc, a component made of a portion of human immunoglobulin D and G4.

Phillips-Medisize , in collaboration with its customer, Ascendis Pharma, was recently recognized with a third industry award for the Skytrofa Auto-Injector. The Red Dot Award recognizes the drug delivery device's exceptional design and positive impact. The device is designed to enable convenient and efficient preparation and delivery of Skytrofa, a growth hormone treatment for children. The Red Dot jury praised the device for its ease of use and optics, as well as convenient and safe process control with reduced pharmaceutical waste. Traditionally, children requiring growth hormone treatments endured daily injections that often led to nonadherence to therapy, due to pain and/or bruising with injection as well as fear of needles. The introduction of the Skytrofa Auto-Injector has reduced the frequency of injections to just once a week, while using a discreet needle. The device previously received the Pharmapack Health Product Award for Patient-Centric Design in May 2022, and the Parenteral Drug Association (PDA) Innovation Award in October 2022. How the Skytrofa Auto-Injector works Skytrofa is a lyophilized powder available in a single-dose, dual-chamber, prefilled cartridge containing lonapegsomatropin-tcgd in one chamber and water in the other. The cartridge is designed for subcutaneous injection once a week with the Skytrofa Auto-Injector. The delivery device provides a fully automated reconstitution of the lyophilized drug product, followed by a manual mixing step controlled by the device. The device automatically delivers the drug product after inserting the injection needle into the skin. Built-in electronics and software assist the user during the entire preparation and injection sequence and confirm that the full dosage is delivered. By making the device reusable and rechargeable, Phillips-Medisize and Ascendis Pharma have eliminated the waste of disposable injector pens and batteries with a full charge lasting four weeks, with one weekly injection. The life of the Auto-Injector is roughly four years or 210 injections. John Christensen, senior project manager in contract development at Philips–Medisize, recently gave MD+DI a demonstration of how the Skytrofa Auto-Injector works (see video below). Design considerations for the Skytrofa Auto-Injector Paul Erik Fabricius, director of front-end innovation at Phillips-Medisize, said there were several key design considerations that went into this device because of the intended user population (parents of young children requiring growth hormone therapy). The team wanted the design to be visually appealing for children and found that the white and bright green colors of the device made it more pleasing for this user population. "We wanted to facilitate this very complex preparation procedure in a simple and safe manner," Fabricius told MD+DI . "That means both the physical handling of the device, for example it needs to stand up during the reconstitution to ensure that no drug is lost out through the needle, but you also need to have a nice grip when you do the injection." Image courtesy of Phillips-Medisize, a Molex company Phillips-Medisize conducted multiple usability studies with more than 300 participants that helped inform the design of the device. The participants included a mix of experienced and unexperienced users, trained and untrained users, healthcare providers and caregivers. It was especially important to test the device with unexperienced, untrained users, Fabricius said, to ensure that it was as intuitive and simple to use as possible. "That's a challenge because, if you imagine, when you see the device, you don't have any references because you've never seen a device like that," he said. "So, you start without any prior knowledge about how to use it, so basically anything can go wrong. But we've tested that with naive and untrained users, and they have all successfully received the dose." Some of the users in these testing groups thought the device was actually needle-free because the 31-gauge needle is so thin, they didn't even feel it. Image courtesy of Phillips-Medisize, a Molex company In addition to extensive usability studies, Fabricius said it was crucial to maintain an open dialogue with the customer throughout the design and development phase of the device. "That was a key thing for success here because in the process things popped up where we needed to change things," he said. "For example, that manual mixing step John showed you, in the beginning we didn't think that was necessary. But it was simply due to the fact that we could see that the drug was not homogenous after the stable mixing step."

COPENHAGEN, Denmark, Aug. 24, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that Jan Mikkelsen, President & Chief Executive Officer, and Scott Smith, Executive Vice President & Chief Financial Officer, will participate in three upcoming investor conferences. Details: EventCiti’s 18th Annual BioPharma ConferenceLocationBoston, MADateThursday, September 7, 2023Time2:40-3:25 p.m. Eastern Time / 11:40 a.m-12:25 p.m. Pacific Time Event2023 Wells Fargo Healthcare ConferenceLocationBoston, MADateFriday, September 8, 2023Time8:00-8:35 a.m. Eastern Time / 5:00-5:35 a.m. Pacific Time EventMorgan Stanley 21st Annual Global Healthcare ConferenceLocationNew York, NYDateTuesday, September 12, 2023Time12:55-1:25 p.m. Eastern Time / 9:55-10:25 a.m. Pacific Time A live webcast of the events will be available via the Investors & News section of the Ascendis Pharma website at investors.ascendispharma.com. A webcast replay will also be available on this website shortly after conclusion of the event for 30 days. About Ascendis Pharma A/SAscendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Germany (Heidelberg, Berlin and Munich) and the United States (Palo Alto and Redwood City, California, and Princeton, New Jersey). Please visit ascendispharma.com to learn more. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ ability to apply its TransCon platform to build a leading, fully integrated, global biopharma company, and (ii) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including inflation, the effects on its business from the worldwide COVID-19 pandemic and ongoing conflicts such as that in the region surrounding Ukraine and Russia. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 16, 2023 and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are trademarks owned by the Ascendis Pharma group. © August 2023 Ascendis Pharma A/S. Investor Contacts: Media Contact:Tim Lee Melinda BakerAscendis Pharma Ascendis Pharma+1 (650) 374-6343 +1 (650) 709-8875tle@ascendispharma.com media@ascendispharma.comir@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com