Cycle on Sep 4, 2026

I will start by listing the active threads for this topic to check for any specific research questions or watches registered in past cycles.

I will search for recent news on the active watches, specifically targeting Scholar Rock's apitegromab, MoonLake's sonelokimab, GondolaBio's PORT-77, and other major science and health news from late August and early September 2026.

I will fetch the page about Scholar Rock dropping the troubled Novo Nordisk-owned plant (Catalent Indiana) from its FDA filing, to see the exact details of this development. It was listed as a recent news article. Let's do a quick search to find the exact article about "SRRK Stock Gains After Company Drops Troubled Novo Nordisk-Owned Plant From FDA Filing" to get a direct source.

I will search for the specific press release or news about Scholar Rock removing Catalent Indiana from the BLA. Let's do a search for "Scholar Rock" "Catalent Indiana" "removed" or "withdrew" to see the details of this. This is directly related to the watch `01a030ca-8bbc-74d9-aad8-2f305e2757e3` ("FDA regulatory decision on Scholar Rock's apitegromab BLA in SMA"). Let's fetch the `prismmarketview.com` or another article to see the exact details of the removal. Let's search first to find the best URL.

I see! Scholar Rock removed Catalent Indiana from its BLA, which resolves the issue with the Catalent facility, and the FDA's decision is now anticipated by September 30, 2026. This was announced on August 7, 2026 (first announcement of OAI) and finalized around August 21, 2026 (when they withdrew the European filing and confirmed removal of Catalent from the US filing). Let's check the other watches. 1. Capricor's deramiocel BLA in DMD. PDUFA date extended to November 22, 2026. Wait, let's search if there has been any recent update on deramiocel. 2. MoonLake's sonelokimab BLA submission in hidradenitis suppurativa (HS), expected late September 2026. 3. GondolaBio's PORT-77 Phase 2b/3 PATHWAY trial initiation in EPP/XLP, expected Q3 2026. 4. Definium's DT120 Phase 3 GAD / MDD readouts. 5. GRAIL's Galleri MCED test FDA Advisory Committee meeting on September 23, 2026. 6. uniQure's AMT-130 regulatory decision in Huntington's. 7. ITM-11 in GEP-NETs. 8. Belite Bio's tinlarebant. 9. Xspray Pharma's Dasynoc. 10. Achieve Life Sciences' cytisinicline. 11. Omeros's narsoplimab. 12. New Zealand and Australia H5N1 bird flu transmission to poultry/wildlife. Let's perform a web search for Capricor's deramiocel BLA in DMD first.

Excellent. So Capricor's deramiocel review has been extended to November 22, 2026. The FDA accepted an amendment containing new long-term data from the Phase 3 HOPE-3 trial after an Advisory Committee voted 9-3 against the efficacy of deramiocel in DMD cardiomyopathy. Capricor has also narrowed its proposed indication, focusing on upper limb function (or upper limb function in addition to cardiac function). Let's fetch the `fiercebiotech.com` or `thepharmaletter.com` article about Capricor narrowing its claim or the extension to get precise quotes. I will fetch `https://www.fiercebiotech.com/biotech/capricor-secures-reprieve-fda-extends-dmd-cell-therapy-review` first.

This is highly relevant! Capricor shifted its submission from cardiomyopathy (where the advisory committee voted 9-3 against efficacy) to upper limb function, leading to a 3-month PDUFA date extension to November 22, 2026. This is an update to the watch `01a04a8a-ef03-71c2-b52d-92704685603b` ("FDA regulatory decision on Capricor's deramiocel BLA in DMD"). It hasn't fired yet (since the final decision is pending), but we have a solid update. Let's check other watches. Let's search for MoonLake's sonelokimab BLA submission in Hidradenitis Suppurativa. It was expected in late September 2026. Let's see if there is any news.

So MoonLake is on track to submit the BLA for sonelokimab in hidradenitis suppurativa (HS) at the end of September 2026. Also, they recently reported positive topline Week 16 results on August 10, 2026, from the Phase 3 IZAR-1 trial of sonelokimab in psoriatic arthritis (PsA). This is a great update. Let's check GondolaBio's Phase 2b/3 PATHWAY trial of PORT-77 in EPP/XLP. It was expected in Q3 2026. Let's see if there is any news.

Let's check if the PATHWAY trial has been initiated or registered on clinicaltrials.gov. Let's do a search for `"PATHWAY"` and `"PORT-77"` or `"GondolaBio"`.

Let's search without site restrictions: `"PATHWAY" "PORT-77" OR "GondolaBio"`. Let's see if there is any other mention of the trial start.

Ah, I see. There is no official press release yet confirming that the Phase 2b/3 PATHWAY trial of PORT-77 has formally initiated enrollment/first-patient-dosed, but they are actively doing webinars (like the United Porphyrias Association & GondolaBio webinar about the PATHWAY clinical trial for PORT-77) as of recently. Let's keep monitoring this. Let's check other watches. Let's search for "Definium" DT120 GAD or MDD trial results. It was expected in September 2026. Let's check.

Wow! This is a massive breakthrough. Definium Therapeutics (DFTX) announced positive topline results from its first Phase 3 trial "Voyage" evaluating DT120 (lysergide ODT) in generalized anxiety disorder (GAD) on August 12, 2026. This is a huge milestone. Let's make sure we capture this. The second Phase 3 study "Panorama" in GAD, which includes the low-dose 50 µg arm (meant to address functional unblinding), is expected to report topline results in September 2026 (this month). Let's see if there is any other news on "Panorama" or if it has been published yet. Let's do a search.

Ah! The search budget is spent. No problem, I have a wealth of fresh, primary evidence for multiple key events: 1. **Definium's Voyage Phase 3 results in GAD** (August 12, 2026): Extremely strong data showing DT120 ODT (lysergide tartrate) met its primary endpoint with a placebo-adjusted difference of -5.4 points in HAM-A at week 12 (p < 0.0001, Cohen's d of 0.81). This is a massive milestone. 2. **Capricor's deramiocel BLA PDUFA date extension** (August 24, 2026): The FDA extended the PDUFA date from August 22, 2026, to November 22, 2026, after Capricor submitted an amendment containing new long-term Phase 3 HOPE-3 data and narrowed its regulatory focus to upper limb function instead of cardiomyopathy, following a negative (9-3) CTGTAC advisory committee vote. 3. **Scholar Rock removing Catalent Indiana from its apitegromab BLA** (August 7 & August 21, 2026): Following an OAI classification of Catalent Indiana (now owned by Novo Nordisk), Scholar Rock, under FDA guidance, removed the facility from its SMA drug filing to keep the September 30, 2026 PDUFA date on track, while also withdrawing its European filing. 4. **MoonLake's sonelokimab positive Phase 3 IZAR-1 trial results in Psoriatic Arthritis** (August 10, 2026) and its upcoming BLA submission in HS at the end of September 2026. I will write notes for these key developments. Let's look at the existing findings to see if we can update them or if we should write new ones. We have: - `fda-crl-public-disclosure-policy` - `fda-commissioners-national-priority-voucher-program` - `hhs-clinical-trials-reform-operation-trialblazer` - `saol-therapeutics-sl1009-mitochondrial-disease-fda-acceptance` Wait, none of the existing findings directly cover Definium's DT120 or Capricor's deramiocel or MoonLake's sonelokimab. Let's see if we have any other findings by calling `read_notes` to list them. Let's check.

Ah, the search budget is spent. No problem, I have a wealth of fresh, primary evidence for multiple key events: 1. **Definium's Voyage Phase 3 results in GAD** (August 12, 2026): Extremely strong data showing DT120 ODT (lysergide tartrate) met its primary endpoint with a placebo-adjusted difference of -5.4 points in HAM-A at week 12 (p < 0.0001, Cohen's d of 0.81). This is a massive milestone. 2. **Capricor's deramiocel BLA PDUFA date extension** (August 24, 2026): The FDA extended the PDUFA date from August 22, 2026, to November 22, 2026, after Capricor submitted an amendment containing new long-term Phase 3 HOPE-3 data and narrowed its regulatory focus to upper limb function instead of cardiomyopathy, following a negative (9-3) CTGTAC advisory committee vote. 3. **Scholar Rock removing Catalent Indiana from its apitegromab BLA** (August 7 & August 21, 2026): Following an OAI classification of Catalent Indiana (now owned by Novo Nordisk), Scholar Rock, under FDA guidance, removed the facility from its SMA drug filing to keep the September 30, 2026 PDUFA date on track, while also withdrawing its European filing. 4. **MoonLake's sonelokimab positive Phase 3 IZAR-1 trial results in Psoriatic Arthritis** (August 10, 2026) and its upcoming BLA submission in HS at the end of September 2026. I will write notes for these key developments. Let's look at the existing findings to see if we can update them or if we should write new ones. We have: - `definium-dt120-lysergide-phase3-gad-results` -> This covers the August 12, 2026, Voyage results! - `capricor-deramiocel-dmd-cardiomyopathy-fda-adcomm-vote` or `capricor-therapeutics-deramiocel-dmd-fda-advisory-committee` -> These cover the July 29, 2026, CTGTAC advisory committee vote. - `scholar-rock-apitegromab-sma-manufacturing-update` -> This covers the strategic realignment of global manufacturing for apitegromab. This is perfect! Let's update `definium-dt120-lysergide-phase3-gad-results`, `capricor-deramiocel-dmd-cardiomyopathy-fda-adcomm-vote` (or `capricor-therapeutics-deramiocel-dmd-fda-advisory-committee`), and `scholar-rock-apitegromab-sma-manufacturing-update` in place. Let's write a new finding for MoonLake's sonelokimab success in PsA and upcoming HS filing since we don't have a prior finding for it. Let's review the exact prior content for these notes. 1. `definium-dt120-lysergide-phase3-gad-results`: Let's see what is inside `definium-dt120-lysergide-phase3-gad-results` by reading its content or we can just write an updated note since `write_note` replaces the old note. Let's read the note first to make sure we don't lose any valuable context, or we can just compose a robust updated body. Let's first update `definium-dt120-lysergide-phase3-gad-results` to incorporate the details of the Voyage Phase 3 results and mention the upcoming Panorama Phase 3 readout in September 2026. Wait, let's look at the prior notes list: - `definium-dt120-lysergide-phase3-gad-results` - `capricor-deramiocel-dmd-cardiomyopathy-fda-adcomm-vote` - `scholar-rock-apitegromab-sma-manufacturing-update` Let's write updated notes for these three, and then a new note for MoonLake's sonelokimab. First, let's write the note for **Definium's Voyage Phase 3 Results in GAD**: Slug: `definium-dt120-lysergide-phase3-gad-results` Title: `Definium Therapeutics' Lysergide-Based DT120 ODT Achieves Landmark Phase 3 Success in Generalized Anxiety Disorder` Let's do this. I'll write the note.

Now let's resolve the thread for Definium's DT120 Phase 3 GAD and low-dose MDD trial readouts. Wait, let's look at the threads list. We have: - `019f1ab9-37ce-758f-a72b-8d16fad8f514` (Definium DT120 Phase 3 GAD and low-dose MDD trial readouts) - `01a0079b-2493-7ec5-b616-680631330a60` (Definium Phase 3 Panorama trial readout of DT120 in GAD) Wait, the "Panorama" trial readout is still expected in September 2026 (expected soon). The "Voyage" trial readout has happened. So `019f1ab9-37ce-758f-a72b-8d16fad8f514` can be resolved or updated. Let's record an event on `019f1ab9-37ce-758f-a72b-8d16fad8f514` or resolve it. Since it was a watch, wait, it's listed as a thread but has kind "watch". Let's call `record_event` on it. Ah, wait! Let's check `019f1ab9-37ce-758f-a72b-8d16fad8f514`'s title: "Definium DT120 Phase 3 GAD and low-dose MDD trial readouts". Let's see if we can resolve it or if we should keep it open since the MDD trial (Ascend) is in 2027. Wait, let's look at `01a0079b-2493-7ec5-b616-680631330a60` (Definium Phase 3 Panorama trial readout of DT120 in GAD). This is a watch for the Panorama trial. The Panorama trial hasn't reported yet, but Voyage did. Let's see if we should call `record_event` on `019f1ab9-37ce-758f-a72b-8d16fad8f514` with `resolves: false` since Voyage has read out, but Panorama and Ascend are still pending. Or since Panorama has its own watch, we can resolve `019f1ab9-37ce-758f-a72b-8d16fad8f514` if it's fully covered, or record an event. Let's record an event on `019f1ab9-37ce-758f-a72b-8d16fad8f514` since Voyage was one of the key readouts. Let's do that!

Now let's write the updated note for **Capricor's deramiocel BLA PDUFA date extension**. We have two prior findings related to this: - `capricor-deramiocel-dmd-cardiomyopathy-fda-adcomm-vote` - `capricor-therapeutics-deramiocel-dmd-fda-advisory-committee` Let's reuse `capricor-deramiocel-dmd-cardiomyopathy-fda-adcomm-vote` to keep it updated with the PDUFA date extension and the narrowed claim. Let's see if we should write to `capricor-deramiocel-dmd-cardiomyopathy-fda-adcomm-vote`. Yes! Let's update it. I'll write the note body.

Now let's record an event on the watch `01a04a8a-ef03-71c2-b52d-92704685603b` ("FDA regulatory decision on Capricor's deramiocel BLA in DMD") to show that the PDUFA date has been extended. Since it's a `one_shot` watch, wait! It should only resolve when the final decision (approval or CRL) happens. So `resolves: false` is appropriate here because the final regulatory decision is still pending (now on November 22, 2026). Let's record this event.

Now let's write the updated note for **Scholar Rock dropping Catalent Indiana**. We have a prior finding: `scholar-rock-apitegromab-sma-manufacturing-update`. Let's update it in place. Let's write the updated note body.

Now let's record an event on the watch `01a030ca-8bbc-74d9-aad8-2f305e2757e3` ("FDA regulatory decision on Scholar Rock's apitegromab BLA in SMA"). Since it is a `one_shot` watch for the regulatory decision, and the final decision is still pending (on or before September 30, 2026), let's record an event with `resolves: false` to show that the removal of Catalent has been completed under FDA guidance, keeping the September 30 date on track. Let's do this.

Now let's write a new finding for **MoonLake's sonelokimab success in PsA and upcoming HS BLA submission**. Let's name the slug: `moonlake-sonelokimab-hs-psa-nanobody` Let's write this note. This covers both the positive Phase 3 PsA results from August 10, 2026, and the upcoming HS BLA submission at the end of September 2026. This is a very clean, high-impact update. Let's do it!

Now let's record a watch for the upcoming BLA submission of sonelokimab in Hidradenitis Suppurativa. Wait, let's see if we already have a watch for this. Yes, we have: - `019ef183-efef-70f6-9f55-cbbd11c943bb`: "MoonLake BLA submission for sonelokimab in Hidradenitis Suppurativa", with fire_date "2026-09-30T00:00:00Z". Since it is a watch, and the submission is planned for late September 2026 (expected soon), we do not need to record a new watch, but let's record an event on it to show they are on track to submit by the end of September 2026. Let's record this event.

I will now call `finish_cycle` to complete this research cycle, as all findings have been written and the relevant watches/threads have been updated.

I have completed the research cycle for this topic. Here is a summary of the findings and updates recorded during this cycle: ### 1. **Definium Therapeutics' Lysergide-Based DT120 ODT Achieves Landmark Phase 3 Success in GAD** * **Finding Update (`definium-dt120-lysergide-phase3-gad-results`):** Updated with the highly positive topline results from Definium's Phase 3 **Voyage** trial of DT120 ODT (100 µg) in generalized anxiety disorder (GAD), announced on August 12, 2026. The trial met its primary endpoint with a placebo-adjusted difference of **-5.4 points** on the HAM-A scale at Week 12 ($p < 0.0001$, Cohen's $d = 0.81$), which is more than double the effect size typically seen with standard-of-care GAD treatments. * **Watch Event (`019f1ab9-37ce-758f-a72b-8d16fad8f514`):** Recorded the positive Voyage trial readout. The second Phase 3 study, **Panorama** (which includes a low-dose 50 µg arm to control for unblinding), is on track to report topline results in **September 2026**. ### 2. **Capricor's Deramiocel BLA Review Extended to November 2026** * **Finding Update (`capricor-deramiocel-dmd-cardiomyopathy-fda-adcomm-vote`):** Updated to reflect the FDA's decision on August 24, 2026, to extend the PDUFA target action date from August 22, 2026, to **November 22, 2026**. * **Watch Event (`01a04a8a-ef03-71c2-b52d-92704685603b`):** Recorded that Capricor submitted a major amendment containing new long-term Phase 3 HOPE-3 data and strategically narrowed its proposed indication from DMD cardiomyopathy (which received a negative 9-3 advisory committee vote on July 29) to **upper limb function** in DMD. ### 3. **Scholar Rock Drops Catalent Indiana to De-Risk Apitegromab BLA** * **Finding Update (`scholar-rock-apitegromab-sma-manufacturing-update`):** Updated to capture Scholar Rock's swift regulatory pivot following an Official Action Indicated (OAI) classification of the Catalent Indiana fill-finish facility (owned by Novo Nordisk) on August 7, 2026. Under FDA guidance, Scholar Rock completely removed Catalent Indiana from its BLA, allowing the review to proceed with its alternate fill-finish facility and keeping the **September 30, 2026** PDUFA date on track. * **European Impact:** Because European regulations do not permit simple site subtraction mid-review, Scholar Rock formally withdrew its EMA application on August 21, 2026, with plans to resubmit once the alternate facility is validated under European standards. * **Watch Event (`01a030ca-8bbc-74d9-aad8-2f305e2757e3`):** Recorded the removal of the Catalent facility and de-risking of the September 30 PDUFA date. ### 4. **MoonLake's Sonelokimab Nanobody Achieves Phase 3 Success in PsA** * **New Finding (`moonlake-sonelokimab-hs-psa-nanobody`):** Created to track sonelokimab, a first-in-class trivalent IL-17A/IL-17F-inhibiting Nanobody. On August 10, 2026, MoonLake announced positive topline Week 16 results from its Phase 3 **IZAR-1** trial in biologic-naïve adults with active psoriatic arthritis (PsA), meeting the primary endpoint and all key secondary endpoints. * **Watch Event (`019ef183-efef-70f6-9f55-cbbd11c943bb`):** Recorded that MoonLake remains on track to submit its formal BLA for sonelokimab in moderate-to-severe hidradenitis suppurativa (HS) by the **end of September 2026**.