ADDED
The number of outstanding shares of the registrant s common stock as of February 20, 2026 was 158,591,491 par value $0.01 per share, outstanding.
This number does not include 28,331,779 shares of common stock issuable upon the exercise of pre-funded warrants outstanding as of February 20, 2026 (which are immediately exercisable at an exercise price of $0.01 per share of common stock, subject to beneficial ownership limitations).
BUSINESS Overview and Strategy Our purpose is to bring the power of biotherapeutics to the whole body, including the brain, by discovering, developing, and delivering medicines for people living with serious diseases.
Historically, the blood-brain barrier has been a major challenge to the development of medicines for diseases of the central nervous system.
We have invented, developed, and validated a proprietary technology, called the TransportVehicle TM ("TV"), to address the blood-brain barrier challenge and enable a new class of barrier-crossing therapeutics.
The biologics license application ("BLA") for tividenofusp alfa is under priority review for accelerated approval by the U.S.
Food and Drug Administration ("FDA"), and we have established commercial readiness in anticipation of the Prescription Drug User Fee Act (PDUFA) target action date of April 5, 2026.
Our TV-enabled clinical development portfolio also includes DNL126 (ETV:SGSH) for mucopolysaccharidosis IIIA ( MPS IIIA , or Sanfilippo syndrome type A), DNL593 (PTV:PGRN) for frontotemporal dementia-granulin ( FTD- GRN ), DNL628 (OTV:MAPT) for Alzheimer s disease, and DNL952 (ETV:GAA) for Pompe disease.
Key elements of our strategy include: 1) Discover: Advance a new class of barrier-crossing therapeutics by leveraging our TV platforms and deep expertise in blood-brain barrier biology to enhance the delivery of biotherapeutics to the brain and throughout the body.
Our goals are to (i) deliver two growing commercial brands, tividenofusp alfa (ETV:IDS) for Hunter syndrome and DNL126 (ETV:SGSH) for Sanfilippo syndrome type A, establishing the foundation for a durable rare disease franchise; (ii) achieve five clinical proof-of-concept readouts across our portfolio, including programs in Alzheimer s disease (ATV:Abeta and OTV:MAPT), FTD- GRN (PTV:PGRN), Pompe disease (ETV:GAA), and Parkinson s disease (LRRK2 inhibitor); and (iii) advance four to six additional TV-enabled programs into the clinic ( Figure 1) .
REMOVED
The number of outstanding shares of the registrant s common stock as of February 21, 2025 was 145,215,391 par value $0.01 per share, outstanding.
This number does not include 26,046,065 shares of common stock issuable upon the exercise of pre-funded warrants outstanding as of February 21, 2025 (which are immediately exercisable at an exercise price of $0.01 per share of common stock, subject to beneficial ownership limitations) sold in the registrant s private placement in February 2024.
BUSINESS Overview and Strategy Our goal is to discover, develop, and deliver a new class of barrier-crossing therapeutics and to defeat degeneration.
Historically, the blood-brain barrier ("BBB") has been a major challenge to the development of medicines for diseases of the central nervous system ("CNS").
We have invented, developed, and validated a proprietary technology, called the TransportVehicle TM ("TV"), to address the BBB challenge.
We plan to submit a biologics license application ("BLA") for tividenofusp alfa under the FDA's accelerated approval pathway in early 2025 and we are preparing for a potential commercial launch in late 2025 or early 2026.
Our TV-enabled clinical development portfolio also includes DNL126 (ETV:SGSH) for MPS IIIA (Sanfilippo syndrome) and DNL593 (PTV:PGRN) for frontotemporal dementia-granulin ( FTD-GRN ).
Our small molecule clinical development portfolio includes BIIB122/DNL151 (small molecule LRRK2 inhibitor) for Parkinson s disease; and DNL343 (small molecule eIF2B activator) for amyotrophic lateral sclerosis ( ALS ).
Key elements of our strategy include: 1) Discover and develop a new class of barrier-crossing therapeutics by leveraging our TV platforms and deep expertise in BBB biology to enhance the delivery of biotherapeutics to the brain and throughout the body.
About two-thirds of these diseases affect the CNS; however, the current standard treatment, enzyme replacement therapy, does not address CNS manifestations, even for the lysosomal storage diseases where enzyme replacement therapy is available.