CureDuchenne’s Post

Pharmaxis (ASX: PXS) is pleased to report the FDA has cleared its protocol amendment to add PXS‐5505 to the treatment regimen for myelofibrosis patients who are already using the JAK inhibitor, ruxolitinib. These new patients will form a new combination arm of the Phase 2 clinical trials which reported interim data last month.    Pharmaxis CEO Gary Phillips said, "This study will be run as an extra cohort of the ongoing monotherapy study and therefore we can get a very fast start with no need to set up new trial centres. This combination holds much promise for myelofibrosis patients with the potential to deliver a disease modifying effect."    Click below to view the commentary in full within Pharmaxis' quarterly shareholder update.  A video presentation covering the FDA news and June quarter can also be viewed here: https://lnkd.in/dKGRDJnz   #fibrosis #myelofibrosis #clinicaltrials #fda #bonemarrowcancer 

On Thursday, March 14, Autolus Therapeutics held its Q4 2024 earnings call (press release: https://bit.ly/48YCpGz / presentation: https://bit.ly/49VcGQt) highlighting the recent submission of a Market Authorization Application (MAA) for obe-cel (#CD19 CAR-T) in r/r ALL, while noting that its Nucleus manufacturing facility has successfully passed its first GMP inspection enabling the commercial product supply for obe-cel. Additionally, the Ph1 #CARLYSLE confirmatory trial evaluating obe-cel in severe, refractory SLE, and the Ph1 #MAGNETO trial (https://bit.ly/43jTSba) studying #AUTO6NG (#GD2 CAR-T) in pediatric #glioblastoma have been initiated. Celltelligence provides insights on Autolus’s preparedness for obe-cel potential launch in 2024, while discussing its efforts to accelerate obe-cel’s evaluation in the #autoimmune disease space: https://bit.ly/CT-862354

We are excited to announce FDA acceptance for filing of a new drug application (NDA) for rivoceranib in combination with camrelizumab as a first-line treatment for unresectable hepatocellular carcinoma (uHCC). This news brings us one step closer to delivering on our commitment to elevate outcomes for patients. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of May 16, 2024. The NDA is supported by clinical data from the phase 3 CARES 310 study (NCT03764293), in which rivoceranib plus camrelizumab demonstrated statistically significant and clinically meaningful prolonged overall survival and progression-free survival, and improved overall response rate versus sorafenib, a standard first-line treatment for uHCC. “With the FDA’s acceptance of our new drug application, we are pleased to reach another milestone in the development of this combination therapy,” said Saeho Chong, Elevar chief executive officer.   Learn more. https://lnkd.in/ehhzZwjt #ElevatingOutcomes #ElevarPressRelease

Congratulations Pliant Therapeutics on the initiation of BEACON-IPF, a Phase 2b clinical trial of bexotegrast in patients with idiopathic pulmonary fibrosis (IPF). #BIBrands BEACON-IPF is a 52-week, multinational, randomized, dose-ranging, double-blind, placebo-controlled Phase 2b trial evaluating bexotegrast at doses of 160 mg or 320 mg. #clinicaltrialname #IPF #naming

Early Phase 2a data from Pliant Therapeutics INTEGRIS-PSC trial look encouraging for people with PSC. This trial had a placebo control group with a 3:1 randomisation, so these interim results are great news for those who took part. We look forward to furture results from INTEGRIS-PSC https://lnkd.in/gryNPTtu #LetsBeatPSC #Hope #PrimarySclerosingCholangitis #PSC

On August 24th, the FDA approved Tyruko (natalizumab-sztn), the first biosimilar to Tysabri (natalizumab) injection for the treatment of adults with relapsing forms of multiple sclerosis (MS). Tyruko, like Tysabri, is also indicated for inducing and maintaining clinical response and remission in adult patients with moderately to severely active Crohn’s Disease (CD) with evidence of inflammation who have had an inadequate response to, or are unable to tolerate, conventional CD therapies and inhibitors of TNF-α (tumor necrosis factor, a substance in your body that causes inflammation). Read more: https://ow.ly/lgjh50PEswL #biosimilar

We are excited to announce today that the first patient has been dosed in the multiple ascending dose (MAD) study of the Phase 1a/b clinical trial of NM26 for the treatment of moderate-to-severe atopic dermatitis #atopicdermatitis (AD). The MAD study in AD patients is a planned continuation of the Company’s ongoing single ascending dose (SAD) portion of the Phase 1a/b trial in healthy volunteers, being conducted in collaboration with Asia-regional partner Kaken Pharmaceutical.    NM26 inhibits three key pathways involved in the pathophysiology of AD. While standard of care blocks IL-4 and IL-13 to reduce inflammation, NM26-2198 also blocks IL-31, thereby targeting an additional independent pathway that contributes to inflammation and itch. We believe that NM26 has the potential to deliver faster relief from itch and more pronounced improvement of skin lesions, providing a more effective therapeutic option for patients.    To learn more, read our press release: https://lnkd.in/eGbzKtrT   #FirstPatient #ClinicalTrials #Inflammation 

AdAlta Ltd (ASX:1AD) has received results of dose simulation studies that support the potential efficacy of its i-body drug candidate AD-214 in fibrotic disease at its planned intravenous dose regime. The simulations also highlight the possibility that AD-214 could be delivered subcutaneously at lower doses, further enhancing the potential of the product.  Combined, the results of the studies — supporting both the potential efficacy of target intravenous doses of AD-214 and that subcutaneous administration could also be effective — reduce Phase II risk and significantly enhance AdAlta’s partnering potential. More at #Proactive #ProactiveInvestors #ASX #1AD http://ow.ly/e37h104Vv5M

Redx Pharma PLC (AIM:REDX) said it has kicked off a phase I clinical trial for RXC008, a 'first-in-class' ROCK inhibitor treatment targeting fibrostenotic Crohn's disease. This study aims to assess the safety and pharmacokinetic profile of the drug - in other words, its activity in the body over some time, including the processes by which RXC008 is absorbed, distributed in the body, localised in the tissues, and excreted. It will be assessed in healthy volunteers with the results from the study expected by the end of this year. Chief executive Lisa Anson told investors: "We are delighted to confirm that the first participant has been dosed in the RXC008 phase I clinical study. "RXC008 is a potential first-in-class treatment for patients with fibrostenotic Crohn's disease, a debilitating condition where successive surgeries are the only treatment option available today. More at #Proactive #ProactiveInvestors #AIM #REDX #Crohnsdisease #treatment http://ow.ly/SBp8105k03L

📒 INFEX Therapeutics announced that it has successfully completed dosing in its Phase I study for RESP-X, the Company's new anti-virulence therapy to treat Pseudomonas aeruginosa (Pa) infections in non-cystic fibrosis bronchiectasis (NCFB) patients. ✔ No serious or adverse events ("SAEs") or infusion reactions were reported, and pharmacokinetics (PK) was dose linear across all four dose escalations. RESP-X shows a long 30-day half-life, and the potential for a three-monthly dosing interval in patients. 🔗 https://lnkd.in/eCTXb6Sh