Arrowhead Research « Terug naar discussie overzicht

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Hulskof schreef op 4 mei 2021 23:07:

Enac data (CF) in juni.

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Wil Helmus schreef op 4 mei 2021 22:49:

Dirk heeft weinig geloof in hif2

For lung (CF) I give $ARWR 75% chance of seeing decent knockdown, 50% acceptable safety.

For cancer, 5% chance of clinically relevant efficacy.

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nelis h schreef op 4 mei 2021 23:16:

[...]

van gezonde vrijwilligers of ook patienten ?

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mvdln schreef op 5 mei 2021 06:37:

In juni en juli alleen mogen we 9 PR’s verwachten:

It’s going to be a very busy time with several potentially important events and readouts. This is especially true for the next few months, so I’m going to focus on events planned for June and July. We expect to do the following in roughly this order: 1. Dose the first patients in the first ARO-APOC3 Phase 2b study, with a second Phase 2b and a Phase 3 study in patients with FCS planned for shortly thereafter; 2. Dose the first patients in the ARO-ANG3 2001 Phase 2b study; 3. Report initial interim results from the ARO-ENaC first-in-human study. This will likely include the Single Ascending Dose safety results in healthy volunteers, gene knockdown data in the cohort of healthy volunteers that received bronchial brushings and lavage, and data from the first cohort of patients with Cystic Fibrosis; 4. Report full 12-month biopsy results from the 2002 open-label study of AROAAT; 5. Report initial interim results from the ARO-HSD first-in-human study; 6. Present preclinical data on ARO-DUX4 at the FSHD Society International Research Congress; 7. Report initial interim results from the ARO-HIF2 first-in-human study; 8. File a CTA for ARO-DUX4 and potentially host a KOL webinar to discuss the disease, the market opportunity, and the potential development path; and, 9. Announce additional programs in the pulmonary space that are already deep into preclinical development and in IND-enabling stage.

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mvdln schreef op 5 mei 2021 06:28:

Juist begonnen aan het transcript. Als je er nog in wilt zitten is het nu het moment. Komende 3 maanden zullen bepalen hoe groot ARWR kan of gaat worden:

We expect a data-rich next couple months, including: ARO-HSD data in NASH patients and those at risk of having NASH, ARO-AAT data in patients with AAT liver disease, ARO-ENAC data in healthy volunteers and a small number of CF patients, ARO-HIF2 data in patients with renal cell carcinoma and ARO-DUX4 data in animal models for FSHD. Three of these expected data readouts relate to three cell types that, to our knowledge, have not been successfully addressed by RNAi in humans. It is not big pharma with tens of thousands of employees and hundreds of billions in market value that may be on the cusp of a breakthrough in one of these areas; it is Arrowhead with less than 300 employees and a market value of approximately $7 billion that may be nearing a breakthrough in all three.

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Tom3 schreef op 5 mei 2021 09:00:

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AB! Vooral de laatste zin spreekt boekdelen. Kan me niet voorstellen dat CA niets weet over de inhoud van de data. Hij heeft sowieso een reputatie van underpromising en over delivery. Het strookt in ieder geval niet met het geneuzel van Dirk H. Dit lijkt me een geweldig koopmoment.

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Hulskof schreef op 5 mei 2021 08:31:

Can siRNA Therapeutics Heal the Brain?


As we’ve reviewed in past articles the discovery and history of RNA interference and siRNA as a potent therapeutic modality, it brings us to a fascinating idea. Does siRNA carry the potential to heal the brain? If so, can it treat more than genetic diseases? What about brain tumors and cancers, Alzheimer’s disease, Multiple Sclerosis, stroke, or behavioral conditions? Utilizing siRNA to heal the brain is an up-and-coming space that has recently seen exciting breakthroughs, and effective treatments for many of these conditions may be a reality very soon.

www.oligotherapeutics.org/can-sirna-t...

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mvdln schreef op 5 mei 2021 09:18:

[...]
Stel dat het lukt, en met de nadruk op STEL, om succesvol te zijn in deze indicaties (Solid tumors, Long) dan is het niet de vraag of maar wanneer we de waarde van pakweg een Moderna of Vertex passeren.

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Tom3 schreef op 5 mei 2021 10:52:

The last program for which we expect to have a clinical readout in the near term is ARO-HIF2, which is designed to inhibit the production of HIF-2a to treat clear cell renal cell carcinoma or RCC. We are currently conducting a Phase 1b dose-finding clinical study in three cohorts with advanced clear cell RCC. The study is designed to evaluate the safety of ARO-HIF2, to determine the recommended Phase 2 dose, and to assess pharmacokinetics and preliminary efficacy, based on RECIST, and post-dose tumoral expression of HIF2-alpha and HIF associated genes.

We have completed dosing in two of the three cohorts and should be able to report on those two cohorts in the coming two months. We made a protocol amendment last quarter to add patients to the study. These are heavily pretreated patients with metastatic lesions in different locations, so biopsy collection is challenging. The new patients were added to give us a better chance of having tumor samples that can be processed, evaluated, and analyzed. We will be looking for data suggesting functional delivery to tumors as well as measurable levels of HIF2 knockdown.

Kennelijk bestonden de bestaande deelnemers aan de trial uit de wat meer recht-uit recht-aan nierkanker patiënten. Als je nu een cohort toevoegt dat nog zieker is (gezien waargenomen uitzaaiingen) dan zou het kunnen zijn dat de resultaten bij de eerdere cohorten niet teleurstellend zijn.