Everest Medicines Ltd1952
▲ PositiveTechnologyrelevance
Everest signs exclusive license for late-stage kidney disease drug DMX-200, expanding its renal pipeline.
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Biotech & Genomic Medicine
Everest Medicines LtdTheme Impact 1
Off-coverage companies 1
Dimerix LimitedPrivate▲ Positive
Capitalrelevance
Dimerix secures licensing deal with Everest, providing upfront and milestone payments for DMX-200.
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BioMarin to Book $283M IPR&D Charge, Cutting Q3 EPS by $1.50
BioMarin Pharmaceutical said it will incur an acquired in-process research and development expense of $283 million in the third quarter. As a result, both GAAP and non-GAAP diluted earnings per share for the quarter will be negatively impacted by $1.50. The company's non-GAAP diluted EPS estimate for the quarter stands at $1.25. The charge stems from an acquired in-process research and development asset and weighs on the quarter's reported and adjusted profitability.
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BMRN · Capital · Negative BioMarin will book a $283M acquired IPR&D charge that cuts Q3 GAAP and non-GAAP EPS by $1.50.
United States2
Agios Halts Tebapivat Across All Indications, Leaving Mitapivat as Sole Growth Driver
Agios Pharmaceuticals has discontinued development of tebapivat across all indications, a pipeline setback that leaves the company increasingly dependent on its marketed mitapivat franchise. The company stopped developing the next-generation pyruvate kinase activator in lower-risk myelodysplastic syndromes in May after a phase IIb study failed to meet its predefined threshold for further development, then discontinued it in sickle cell disease in July when phase II data showed improvements in hemoglobin and hemolysis but not enough differentiation from existing PK activators. Mitapivat, marketed as Pyrukynd for PK deficiency and as Aqvesme for thalassemia in the United States and as Pyrukynd for PK deficiency and thalassemia outside the United States, generated $44.7 million in worldwide revenues in the second quarter of 2026, up 259.3% year over year. Agios is also developing mitapivat for sickle cell disease, where its supplemental application received FDA priority review with a final decision expected by Nov. 1, 2026, though Novo Nordisk's investigational etavopivat is advancing toward regulatory submission. Agios shares have declined 29% over the past three months but have gained 14.9% year to date, compared with the industry's 3.3% growth.
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Biotech & Genomic Medicine › Rare Disease Technology
AGIO · Technology · Negative Agios discontinued development of tebapivat across all indications after phase IIb and phase II failures, a pipeline setback leaving it dependent on mitapivat.
AGIO · Capital · Positive Mitapivat (Pyrukynd/Aqvesme) generated $44.7M in Q2 2026 worldwide revenues, up 259.3% year over year.
NVO · Competition · Negative Novo Nordisk's investigational etavopivat is advancing toward regulatory submission, competing with Agios's mitapivat in sickle cell disease.
United States2
Capricor Falls 10% as Deramiocel OLE Data Fails to Ease FDA Concerns
Capricor Therapeutics shares fell about 10% in Monday trading despite positive data from an open-label extension study of deramiocel for Duchenne muscular dystrophy, as investors remained doubtful the candidate will win US FDA approval. A 24-month crossover analysis of HOPE-3 found that patients who began deramiocel after 12 months on placebo slowed upper limb decline by 76% compared to the first year, while patients always on deramiocel showed a similar reduction in rate of decline at both 12 and 24 months. H.C. Wainwright's Joseph Pantginis, who rates Capricor at neutral, said he expects a Complete Response Letter from the FDA for deramiocel, writing that the OLE data strengthens the efficacy story but does not resolve the regulatory uncertainty tied to what occurred during the randomized portion of HOPE-3. Cantor Fitzgerald's Kristen Kluska, who rates Capricor at overweight, was more optimistic, saying the 24-month OLE data reinforce the durability and consistency of the treatment effect and that she leans more toward a potential approval with an attractive risk/reward setup of plus 300% to minus 70%. The OLE analysis was included in a major amendment to the company's BLA, and deramiocel faces a Nov. 22 FDA action date after a late July FDA advisory panel failed to endorse the candidate following briefing documents from agency scientists that called HOPE-3 data into question.
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Biotech & Genomic Medicine › Rare Disease Regulation
CAPR · Regulation · Negative Deramiocel OLE data fails to resolve FDA regulatory uncertainty ahead of the Nov. 22 action date, with analysts expecting a Complete Response Letter.
United StatesInsmed CFO Sara Bonstein to Step Down October 30; Shares Fall 8%
Insmed Inc announced that Chief Financial Officer Sara Bonstein will step down on October 30, sending shares down 8% Monday. Bonstein will continue as CFO through the company's reporting of its third-quarter 2026 financial results and will participate in the earnings call on October 29, while Insmed has begun a search for her successor. She served as CFO for nearly seven years, during which Insmed raised more than $4.2 billion in capital, and the company said her transition is not related to any disagreement over accounting practices, financial statements, internal controls, or operations. Chair and Chief Executive Officer Will Lewis said Insmed is well positioned to reach cash flow positivity next year with a clear path toward sustained top-line growth and bottom-line profitability. The company reaffirmed its full-year 2026 guidance of revenue in the range of $1.25 billion to $1.40 billion for BRINSUPRI and $450 million to $470 million for ARIKAYCE, and will release third-quarter 2026 results on October 29.
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Biotech & Genomic Medicine › Rare Disease Talent
INSM · Capital · Negative CFO Sara Bonstein is stepping down on October 30, a leadership change that sent shares down 8%.
JapanUnited States
European Union▲3
Shionogi to Acquire IntraBio for USD 2.0 Billion, Adding AQNEURSA to Rare Disease Portfolio
Shionogi & Co., Ltd. announced that its Board of Directors approved an agreement to acquire IntraBio Inc., a biopharmaceutical company developing and commercializing therapies for neurodegenerative diseases, for an upfront consideration of USD 2.0 billion payable to IntraBio shareholders. Under the agreement signed on October 5, 2026, IntraBio would become a wholly owned subsidiary of New Jersey-based Shionogi Inc., with the transaction scheduled to close between November 2026 and December 2026, subject to competition-law waiting periods and other customary conditions. The deal would add AQNEURSA (levacetylleucine) to Shionogi's rare disease portfolio; the drug was approved by the FDA in September 2024 for neurological manifestations of Niemann-Pick disease type C and by the European Medicines Agency in January 2026, and on September 18, 2026 it became the first and only FDA-approved treatment for Ataxia in patients with Ataxia-Telangiectasia, for which it is also under EMA review. Shionogi said the acquisition builds on the rare disease foundation it established through its April 2026 acquisition of global rights to edaravone, known as RADICAVA in the U.S. and RADICUT in Japan, and would strengthen its pipeline across Pompe disease, Fragile X syndrome, Jordan's syndrome and early-stage rare neurodegenerative programs. The impact on Shionogi's consolidated financial results for the fiscal year ending March 2027 is currently under review.
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Biotech & Genomic Medicine › Neuroscience & Neurodegenerative ▲Capital
4507.JP · Capital · Positive Shionogi's board approved a USD 2.0 billion acquisition of IntraBio, adding AQNEURSA and rare-disease pipeline assets to its portfolio.
IntraBio Inc. · Capital · Positive IntraBio is being acquired by Shionogi for USD 2.0 billion upfront, delivering consideration to its shareholders.
Global▲3impact 4
CSL Strikes $1.55B Lixudebart Deal With Alentis for Rare Kidney and Liver Diseases
CSL and Alentis Therapeutics have entered an exclusive global partnership to co-develop and co-promote lixudebart for rare kidney, liver, and other diseases. Under the agreement, CSL will arrange an initial payment of $355M to Alentis, which is also eligible for up to $1.2B in commercial milestone payments, bringing the total deal value to $1.55B. The companies will share global profits 55% to CSL and 45% to Alentis once the drug is commercialized. CSL will fund Phase 2 and planned Phase 3 studies of lixudebart in AAV-RPGN while advancing Phase 2 programs in FSGS and PSC. Lixudebart is currently being evaluated in the Phase 2 RENAL trial for AAV-RPGN, a rare autoimmune disease that can cause rapid kidney function loss and irreversible kidney damage.
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