Shots:
Japan's MHLW has granted conditional & time-limited approval to Elevidys for DMD pts (3–8yrs.) with no exon 8/9 deletions in the DMD gene & who are negative for anti-AAVrh74 Abs
Approval was based on extensive clinical data, incl. 2yr. global P-III (EMBARK) trial showing motor function benefits vs external controls; Part 2 data was…
NEWS
Shots:
Following FDA 510(k) clearance, Mentice has launched Ankyras 4.0 for pts-specific flow diverter planning to treat intracranial aneurysms in the US
Ankyras 4.0 introduces DICOM node integration, a redesigned user interface & broader accessibility via online platform & mobile apps, plus its integration with VIST simulation allows virtual case rehearsal to accelerate decision-making &…
Shots:
AbTherx has entered into a multi-program antibody discovery partnership with Spice Biotechnologies to identify antibodies against targets selected by Spice Bio, using AbTherx’s Atlas Mouse technologies
As per the deal, Spice Bio will obtain rights to develop & commercialize antibodies from the collaboration in exchange for research payments, potential clinical & commercial milestones, plus…
Shots:
QIAGEN has entered into a definitive agreement to acquire Genoox, a provider of AI-driven software that allows clinical laboratories to scale & accelerate the analysis of complex genetic tests
As per the deal, Genoox will receive $70M in cash & up to $10M in additional milestone payments
Acquisition adds Franklin, Genoox’s cloud-based platform, to…
Shots:
Genentech has reported 10yr. P-III (APHINITY) trial data assessing Perjeta + Herceptin & CT vs Herceptin + CT vs PBO as an adj. therapy in 4804 pts with operable HER2+ early-stage breast cancer
After 10yrs, trial depicted improved OS, with 91.6% pts alive vs 89.8% on Herceptin + CT & PBO, with sustained invasive…
Shots:
The US FDA has approved IND application of MVX-220 for the treatment of Angelman Syndrome (AS) to initiate P-I/II (ASCEND-AS) trial, with first pts dosing expected in H2’25
The P-I/II trial will evaluate the safety, tolerability, & effectiveness of MVX-220 in both adult & pediatric pts with multiple AS genotypes, incl. rare types like uniparental…
Shots:
The UK’s MHRA has approved Trixeo Aerosphere (budesonide/glycopyrronium/formoterol fumarate or BGF) with HFO-1234ze(E), a next-generation, near-zero global warming potential (GWP) propellant for treating COPD in adults; regulatory review is ongoing in EU, China & other regions
Approval was based on a next-generation propellant clinical development program assessing Trixeo + HFO-1234ze(E) vs Trixeo + HFA-134a,…
Shots:
The P-III (REAL8) basket trial assessed Sogroya (0.24mg/kg, QW or 0.05 mg/kg, QD) in pre-pubertal children with NS, TS, or ISS; SGA-born children were randomized to Sogroya 0.24mg/kg QW or QD dosing (0.035 or 0.067mg/kg)
Trial met its 1EP, showing QW dosing was non-inferior to GH therapy (QD) at Wk. 52, with superior HV…
Shots:
Japan’s MHLW has received MAA for Eylea 8mg (114.3mg/ml for injection) to treat pts with macular edema following RVO (central, branch & hemiretinal) supported by the P-III (QUASAR) trial; regulatory filings are ongoing in other markets
In the trial, Eylea 8mg (Q8W after 3 or 5 monthly doses) met its 1EP at 36wks. with…
Shots:
LENZ has granted Lotus exclusive development, manufacturing, registration & marketing rights of LNZ100 to treat presbyopia in the Republic of Korea & certain Southeast Asian countries incl. Thailand, Philippines, Vietnam, Malaysia, Brunei, Indonesia & Singapore
As per the deal, LENZ will obtain ~$125M in upfront, regulatory & commercial milestones, along with double-digit net sales-based…

