rare diseases

Angelini Pharma acquires Catalyst Pharmaceuticals for $4.1 billion at $31.50 per share, with concurrent patent litigation resolution for rare disease drug Firdapse.

Angelini Pharma to acquire Catalyst Pharmaceuticals for $4.1 billion in all-cash deal, marking entry into U.S. market and strengthening rare disease focus.

Privium Fund Management acquires $5.07M stake in Denali Therapeutics following FDA approval of first commercial drug Avlayah for Hunter syndrome treatment.

BioCryst secures $70M upfront plus $275M milestone payments for navenibart European rights, reinforcing strategy to focus on U.S. commercialization.

ProQR Therapeutics nominates Boehringer Ingelheim's Chief Medical Officer Dr. Lykke Hinsch Gylvin to its Board, pending shareholder approval.

Small-cap stocks outperform with 8.4% monthly gains. Q1 earnings expected to grow 18.4%, nearly double S&P 500's 9.8%, signaling potential breakout opportunities.

Biogen partners with Alloy Therapeutics to advance antisense oligonucleotide therapies using the AntiClastic platform for multiple undisclosed disease indications.

Pharming Group schedules April 2026 investor conferences following positive CHMP opinion for Joenja® leniolisib in APDS treatment.

Agios Pharmaceuticals to report Q1 2026 results April 29 amid FDA accelerated approval pursuit for sickle cell mitapivat and UAE thalassemia approval.

Biogen acquires Apellis Pharmaceuticals for $5.6 billion in cash, gaining two FDA-approved therapies with $689M in 2025 projected sales and mid-to-high teens growth expectations.

Soligenix reported zero 2025 revenue and $11.1M net loss. Company has $7.9M cash, seeking partnerships to fund operations through Q4 2026 pivotal trials.

Boone Capital liquidates entire $13.57M Cogent Biosciences stake despite 372% gain, reallocating to earlier-stage biotech plays like TYRA. Strategic rebalancing in volatile sector.

Biodexa Pharmaceuticals executes 1-for-5 reverse ADR split effective April 6, 2026, to maintain Nasdaq listing compliance with minimum bid price requirements.

Catalyst Pharmaceuticals trades at 20-33% discount to peers with 7.5% FCF yield; 2026 Hetero litigation is binary catalyst determining if shares reach $28-32 or collapse to $16-20.

BioCryst awards 65,850 RSUs to six new employees under Nasdaq inducement rule, vesting over four years starting March 2027.

Palvella Therapeutics closed $230M public offering at $125/share on February 27, 2026, fully exercising underwriter options to fund rare skin disease programs.

Larimar Therapeutics raises $100M through expanded share offering at $5 per share, funding development of lead drug candidate nomlabofusp and operations.

Larimar Therapeutics raises $100M through expanded public offering at $5 per share, funding clinical development of rare disease therapeutic candidate nomlabofusp.

Commodore Capital exits majority Centessa stake, selling 1.85M shares for $46.86M amid 53% stock rally and company's strategic leadership changes.