QXO has raised $830 million through a private placement and priced a public offering of 37.7 million shares at $13.25 per share, generating approximately $500 million to fund its pending acquisition of Beacon Roofing Supply.

The US orphan drug market is projected to surpass $190 billion by 2030, with over 7,300 molecules receiving FDA Orphan Drug Designation to date, of which approximately 17.9% have gained approval.

Medicus Pharma's Phase 2 trial of SKNJCT-003, a non-invasive treatment for basal cell carcinoma (BCC), has randomized over 50% of the targeted 60 patients in the US.

A multinational study of 99 Latin American patients with unresectable hepatocellular carcinoma found only 18% experienced immune-related adverse events (irAEs) with atezolizumab-bevacizumab therapy, significantly lower than clinical trial rates.

Recursion's MEK 1/2 inhibitor REC-4881 demonstrated a median reduction of over 30% in total polyp burden after 12 weeks in patients with familial adenomatous polyposis (FAP), a rare hereditary colorectal cancer syndrome.

Paradigm and Bristol Myers Squibb are partnering to create a new clinical trial model focused on improving patient access, accelerating trial completion, and easing provider burden.

The INAVO120 phase 3 trial demonstrated that inavolisib plus fulvestrant and palbociclib significantly improved progression-free survival (PFS) in patients with HR+, HER2–, PIK3CA-mutated breast cancer.

• The FDA's draft guidance on decentralized clinical trials (DCTs) has been released, aiming to provide a framework for incorporating decentralization into trial designs. • Key challenges identified include Principal Investigator (PI) oversight when using local healthcare providers (HCPs) and ensuring the scalability of data collection in decentralized settings. • The industry seeks clarification on terminology and the integration of AI and machine learning technologies within DCT frameworks to enhance trial efficiency and future applicability. • Experts emphasize that while the guidance is a positive step, continuous feedback and adaptation are crucial to solidify DCTs as a standard component of clinical trial execution.

Recce Pharmaceuticals received patent approval from China's National Intellectual Property Administration for its "Copolymer and Method for Treatment of Bacterial Infection" covering RECCE 327 and RECCE 529 anti-infectives.

Immutep's EFTISARC-NEO Phase II trial demonstrates that 67% of patients achieved near-complete pathologic responses with eftilagimod alpha combined with radiotherapy and pembrolizumab.

The Düsseldorf local division of the Unified Patent Court has dismissed Sanofi and Regeneron's infringement suit against Amgen, allowing continued sales of the PCSK9 inhibitor Repatha across five European countries.

Xenon Pharmaceuticals is progressing its Phase 3 epilepsy program with azetukalner, anticipating topline data from the X-TOLE2 study in the second half of 2025.

NIH researchers have pinpointed AKT2's role in lysosome dysfunction, leading to drusen formation, a key indicator of dry age-related macular degeneration (AMD).

Merck KGaA has initiated a Phase 3 clinical trial, MyClad, to evaluate the safety and efficacy of oral cladribine capsules for treating generalized myasthenia gravis (gMG).

NXP800, a novel GCN2 kinase activator, has been granted orphan drug designation by the FDA for treating ARID1a-deficient ovarian, fallopian tube, and primary peritoneal cancers.

Arrowhead Pharmaceuticals is advancing two RNAi-based therapies, ARO-INHBE and ARO-ALK7, into clinical trials for obesity treatment, targeting novel mechanisms of action.

Aelis Farma's AEF0117 did not meet its primary endpoint of reducing cannabis use to one day or less per week in patients with moderate to severe cannabis use disorder (CUD).

Immunic's IMU-838, an oral DHODH inhibitor, has advanced into a Phase 2 clinical trial targeting post-COVID syndrome, also known as long COVID.

Interim Phase 2 trial results indicate that ifinatamab deruxtecan (I-DXd) demonstrates notable activity against small cell lung cancer (SCLC).

The Alzheimer's disease therapeutic pipeline is expanding beyond amyloid and tau targets, with 32 therapeutics in 48 Phase III trials exploring neuroprotection, neurotransmitters, and inflammation.