Jazz Pharmaceuticals Plc

Significantly increased quality and lower manufacturing costs for long template mRNAs SAN DIEGO, March 26, 2025 /PRNewswire/ -- Primrose Bio, Inc. ("Primrose"), a company developing proprietary therapeutic manufacturing technologies, announces the launch of Prima RNApols™ ExTend, an RNA polymerase engineered to significantly improve manufacturing of long-template mRNA vaccines and therapeutics, including mRNA medicines based on self-amplifying mRNAs. This enzyme shows improved performance, resulting in superior mRNA quality at a lower cost. Prima ExTend outperforms traditional T7 polymerase by generating higher-quality mRNA with lower dsRNA, eliminating a common challenge in mRNA manufacturing and enhancing downstream purification efficiency. Furthermore, Prima ExTend's transcription efficiency results in higher yield using lower amounts of key inputs, which can contribute to lower production costs. "Prima ExTend is a much-needed innovation for the production of long-template mRNA therapeutics, that often struggle with integrity and quality issues that add risk to these programs. Our new enzyme is an important advancement for drug developers and researchers seeking high-integrity, high-yield mRNA," said Helge Zieler, CEO. "This innovation underscores our commitment to providing leading technologies that support the manufacturing of the next generation of mRNA vaccines and therapeutics." Prima RNApols ExTend is available in an in vitro transcription kit for research use applications. GMP-grade Prima RNApols manufactured and released under ISO 13485:2016 certified quality systems are available for clinical and commercial mRNA manufacturing. For more information, please visit . About Primrose Bio, Inc. Primrose Bio is focused on developing and commercializing its integrated end-to-end technology platform for solving complex drug manufacturing and design challenges. Primrose major offerings include Prima RNApols™: improved enzymes for mRNA manufacturing; Pfenex Expression Technology®: a proprietary production system used in six approved products with up to 20x higher yields and unparalleled success in making complex proteins; and PeliCRM197®: a commercially validated CRM197 carrier protein used for conjugate vaccines. Several of the world's leading pharmaceutical companies use Primrose's technologies, including Merck, Jazz Pharmaceuticals, Alvogen, Serum Institute of India, Arcellx, Arcturus and others. For more information, please visit . SOURCE Primrose Bio WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

A phase 3 study evaluating Axsome's Sunosi as a treatment for ADHD has achieved its primary and a key secondary endpoint, the New York City company said. Taking a gamble by purchasing the rights to an underperforming narcolepsy drug may soon begin paying dividends for Axsome Therapeutics.The New York City central nervous system (CNS) specialist has scored in a phase 3 trial testing Sunosi (solriamfetol) on adults with attention deficit hyperactivity disorder (ADHD).The study achieved its primary and key secondary endpoint as Sunosi, compared to placebo, provided “substantial and statistically significant” improvement in ADHD symptoms and a “statistically significant” reduction in overall ADHD severity.In 2022, Axsome paid $53 million up front to acquire Sunosi from Jazz Pharmaceuticals, three years after the Ireland drugmaker scored FDA approval to treat patients with narcolepsy and obstructive sleep apnea (OSA).The companies struck the deal believing Axsome was better equipped to develop the dopamine and norepinephrine reuptake inhibitor (DNRI) for other indications—including major depressive disorder (MDD), binge eating disorder (BED), and excessive sleepiness associated with shift work disorder (SWD).In the ADHD trial, coded Focus, Axsome tested 516 adults in three treatment arms, with patients receiving placebo or Sunosi in a 150 mg dose or a 300 mg dose for six weeks.As measured by the Adult ADHD Investigator Symptom Rating Scale (AISRS), Sunosi dosed at 150 mg provided average reductions from baseline of 17.7 points, compared to 14.3 points for placebo. Overall, Sunosi provided a 45% mean reduction from baseline in ADHD symptoms. With clinical response defined as a 30% improvement from baseline, 54% of patients in the 150 mg arm reached the standard compared to 41% of those on placebo.As for the 300 mg dose of Sunosi, results were numerically superior to placebo, but came up short of statistical significance.The results square up with those produced by Supernus’ Qelbree, “which showed a similar 3.8-point placebo-normalized reduction in AISRS at Week 6,” William Blair analyst Myles Minter, Ph.D., wrote in a note to investors Tuesday, though he warned of the difficulty in making cross-trial comparisons.“The symptom improvements observed with solriamfetol were accompanied by a favorable safety and tolerability profile,” Gregory Mattingly, M.D., president of the American Professional Society for ADHD and Related Disorders, said in a release. “Based on these compelling data, solriamfetol has the potential to be an important new treatment option for adult patients living with ADHD.” Axsome did not address a regulatory filing timeline for Sunosi in the indication. CEO Herriot Tabuteau, M.D., said that the company plans to initiate a trial in pediatric patients this year. Citing FDA guidelines, Minter suggested that Axsome may need to run two separate pediatric trials for both adolescents 13 to 17 years of age and younger kids. Upon the approval of Sunosi six years ago, Leerink Partners analyst Ami Fadia projected potential sales of $500 million by 2023, given its novel mechanism of action and strong efficacy data to back it up.But the treatment was never on that trajectory under Jazz, which reported the drug's sales as $58 million in 2021. Sunosi has shown more promise under the wing of Axsome as 2024 sales reached $94 million, which was an increase of 26% from 2023. Things are happening quickly for Axsome, which Tabuteau founded in 2013. Two months ago, the company gained an FDA approval for migraine treatment Symbravo and saw its market cap surge to $5.3 billion. Axsome has projected Symbravo as a potential blockbuster.Axsome's first approval came in 2022 for major depressive disorder (MDD) treatment Auvelity, which racked up sales of $291 million last year. Potentially building on Auvelity, Axsome is also expected to report data for Sunosi as an MDD treatment from the phase 3 Paradigm study in the first quarter. However, as Minter noted, more than one phase 3 trial is needed for an MDD filing with the FDA.Minter added that the latest ADHD readout “adds to a string of recent wins for the company, including clarity" on a regulatory path to an NDA filing for Alzheimer’s disease treatment AXS-05.

Axsome Therapeutics said the lower dose of its sleep disorder drug Sunosi met the primary endpoint in a late-stage test of adults with ADHD. But Wall Street analysts noted that less impressive results for its higher dose leave room for uncertainty about the company’s label expansion prospects. The 150 mg dose of Sunosi produced an average 17.7-point reduction in an ADHD symptom rating sale called the AISRS at six weeks versus a 14.3-point drop for placebo — a statistically significant result, according to a Tuesday release . The Phase 3 FOCUS trial compared two once-daily doses of Sunosi with placebo in 516 ADHD patients. Participants treated with the 150 mg dose saw an average 45% reduction in their symptoms from baseline to six weeks. The higher 300 mg dose also performed better than placebo in the trial’s primary and secondary endpoints, but the results weren’t statistically significant. Axsome said the higher dose was merely “exploratory.” The company is moving forward with plans for a pediatric trial in ADHD this year, which it will need for an FDA filing. If approved, Sunosi could reach around $300 million in peak sales in ADHD, Leerink analysts said. Axsome’s share price $AXSM dipped around 5% at market open Tuesday. In a note shared Tuesday, TD Cowen analysts said the fact that the higher dose of Sunosi did not perform better than the lower dose in the FOCUS trial “will likely lead to some questions from investors.” But they added that “the totality of the results continue to support that solriamfetol does have activity in this patient population.” The Cowen analysts said that the placebo-adjusted improvement seen with Sunosi is similar to that achieved by Supernus Pharmaceuticals’ ADHD medicine Qelbree in its registrational study. They added that Sunosi could also benefit from a “slightly faster onset of action.” Axsome said Sunosi was well tolerated in FOCUS but didn’t disclose the number of side effects. The drug won FDA approval for certain forms of excessive daytime sleepiness back in 2019. It acts on multiple targets, inhibiting the reuptake of dopamine and norepinephrine while also activating TAAR1 and 5-HT1A. Sunosi was originally developed and marketed by Jazz Pharmaceuticals, before it was divested to Axsome in a 2022 deal featuring $53 million upfront. The US biotech is exploring the drug’s potential in a range of other indications, including major depressive disorder and binge eating disorder.