Upfront Briefing
Year four is where the Huntington's story got complicated. uniQure's AMT-130 slowed decline 44% on cUHDRS at month 48, down from 75% a year earlier and no longer significant, with an accelerated-approval BLA already at the FDA. Lilly, meanwhile, rolled its full TRIUMPH-2 deck into EASD in Milan: 20.8% weight loss in obesity with type 2 diabetes, a Lancet paper, and a Q1 2027 filing pencilled in. Merck finally showed its tulisokibart work in hidradenitis suppurativa at EADV, a second leg for the $10.8B Prometheus buy. Elsewhere, Lundbeck is reportedly circling Xeris (which says it isn't for sale), Oura shelved its up-to-$2.2B IPO while Immix and Pyxis still got follow-ons priced, and four startups raised ~$240M between them.
Tape Action
| Instrument |
Last close |
1D % |
YTD % |
| S&P 500 |
7,670.8 |
(0.2%) |
11.8% |
| Nasdaq 100 |
30,339.3 |
0.2% |
20.4% |
| Russell 2000 |
2,807.9 |
(0.4%) |
11.9% |
| Healthcare (XLV) |
170.7 |
(0.3%) |
9.8% |
| Biotech (XBI) |
156.8 |
0.1% |
29.0% |
| Nasdaq Biotech (NBI) |
7,132.7 |
0.1% |
25.0% |
| Clinical Trials (BBC) |
45.9 |
(0.6%) |
21.9% |
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- Biotech held a sliver of green while broader healthcare slipped: XBI +0.1% against XLV (0.3%), with clinical-stage BBC (0.6%) the laggard.
- Long-end yields kept grinding higher, with the 10-year at 5.25% per AP, even as Brent fell 1.7% to $96.16 on renewed US-Iran talks; consumer confidence at a 12-year low and Nvidia giving up early gains to close (0.7%) left the S&P 500 (0.2%) and Russell 2000 (0.4%) lower while the Nasdaq 100 eked out +0.2%.
- Market data: U.S. cash close Tuesday, 29 September 2026.
The Big 3
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1
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uniQure's AMT-130 benefit fades at year four; QURE opens 41% lower
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- At month 48, AMT-130 was linked to a 44% slowing of Huntington's progression on cUHDRS versus an external control in the 12-patient Phase 1/2 cohort, down from 75% at month 36 and no longer statistically significant; total functional capacity held at 61% slowing (60% at month 36). QURE opened 41% lower at $23.15.
- Why it matters: The BLA filed for accelerated approval earlier this month rests on the month-36 read against the prior external control, so reviewers now weigh a fading primary signal against a stable TFC. Management argues the comparator got healthier as sicker controls dropped out, with more than half of the month-48 control data missing; a post hoc re-run on the prior control shows 54%. Guggenheim flags one outlier driving about a third of the cUHDRS slide. TFC is the confirmatory trial's primary endpoint, which is the bull case in a sentence. The risk is a review division asked to bless a 12-patient, externally controlled dataset whose headline effect shrank 31 points in a year.
- Source: Endpoints
- More: Fierce Biotech; BioPharma Dive
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2
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Lilly's full TRIUMPH-2: retatrutide hits 20.8% weight loss in obesity with T2D
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- At EASD and in The Lancet, retatrutide 12 mg delivered 20.8% mean weight loss at week 80 (efficacy estimand) vs 4.0% on placebo in 1,152 adults with obesity and type 2 diabetes; 34.9% lost at least 25%, and more than half no longer met BMI criteria for obesity. A1C fell 1.4–1.6% from a 7.7% baseline, with no dose response.
- Why it matters: The new information is in the responder tails, and it widens the gap to tirzepatide: 52% on the top dose lost at least 20% of body weight, against 34% at week 72 in a tirzepatide Phase 3. Glycaemia is the soft spot. Only 39–40% on the middle and top doses reached an A1C below 5.7%, versus up to 55.3% on tirzepatide in an earlier trial, which keeps Mounjaro the diabetes-first incumbent. Cardiometabolic markers moved hard, with triglycerides down 39.5% and hs-CRP down 58.3%. Lilly plans to file in Q1 2027. Pressure is arriving from China: TD Cowen said Hengrui and Kailera's triple-G looked better at week 12 in a Phase 1 shown at the same meeting.
- Source: Fierce Biotech
- More: Endpoints; The Lancet
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| 3 | Merck's tulisokibart clears hidradenitis suppurativa Phase 2b at EADV |
- Full Phase 2b data at EADV: 72% of high-dose patients hit HiSCR50 (at least a 50% cut in abscesses and inflammatory nodules) vs 35% on placebo, with both middle and high doses statistically significant. Topline came with Merck's Q2 results, alongside a Phase 2 miss in SSc-ILD.
- Why it matters: A 37-point placebo gap is wider than the deltas behind the approved IL-17s Cosentyx and Bimzelx in their HS Phase 3s, though HS placebo rates swing widely, so cross-trial reads need a discount. For Merck it is the second clinical leg under the $10.8B Prometheus price after positive Phase 3 ulcerative colitis data in June, with the programme now spanning six indications after SSc-ILD was dropped in August. The TL1A class is crowded: Roche's afimkibart and Sanofi/Teva's duvakitug are also in late-stage testing. Phase 3 design and dose selection in HS are the next disclosures to watch.
- Source: Endpoints
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Everything Else that broke
- Beam sued YolTech, its CEO and co-founder (ex-Beam scientist Emma Wang) and US licensee Serapha Bio, alleging stolen base-editing and LNP trade secrets underpin Serapha's AATD candidate SERP-01; Serapha denies the claims. — Endpoints
- Vanda's Hetlioz met its Phase 3 primary endpoint in delayed sleep-wake phase disorder (n=40), advancing sleep onset 37.1 minutes more than placebo; 60% vs 15% shifted by at least 30 minutes, and an sNDA is planned. — PR
- PhRMA named former House Majority Leader Eric Cantor its next president and CEO. — Endpoints
- Fuller ZUPREME-1 data at EASD showed Zealand/Roche's amylin analog petrelintide peaking at 10.7% weight loss at the middle dose over 42 weeks (1.7% placebo), behind the 20.1% for eloralintide from Lilly. — Endpoints
- Enanta laid off 37 staff (of 120 at last count) ahead of a year-end urticaria readout for KIT inhibitor EDP-978 and 2027 infant RSV data for zelicapavir. — Fierce Biotech
- SK pharmteco unveiled a $200M+ CDMO expansion plan over the next five years. — Endpoints
- Invivyd said it plans a Covid filing, setting up a regulatory catalyst. — Endpoints
- Lundbeck has expressed interest in acquiring Xeris (~$1.8B market cap) and is working with advisers; Xeris says it is not for sale. — Bloomberg
- Oura postponed its up-to-$2.2B Nasdaq IPO hours before pricing, citing market uncertainty despite strong demand. — Endpoints
- The US set out rules for 0% tariff exemptions on specialty pharmaceuticals (Section 232 watch). — Fierce Pharma
Deal Flow
| BioBucks 2026 Deal Trackers • Updated weekly ⬇️
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M&A / BD&L
- ARCH and Population Health Partners launched Somnivera with a $10.5M-upfront, ex-China licence to a Jiangsu Nhwa sleep-disorder drug (NewCo BD&L; China-sourced). — Endpoints
- Keros expects a $20M development milestone from Takeda after first patient dosed in elritercept's ELRiSE trial. — PR
VC / Private Financings
- ai3Bio launched with a $48M Series A co-led by UPMC Enterprises and Ziff Capital Partners, joined by Cockrell Interests and Tanis Ventures, for in vivo Th17-depleting antibody and LNP-mRNA autoimmune platforms. — Fierce Biotech
- AnaCardio raised a €62.5M ($70.9M) Series B co-led by Novo Holdings and the Ljungström family office, joined by Helsinn, Pureos Bioventures and Sound Bioventures, funding the 400-patient GOAL-HF2 Phase 2b of oral AC01 in HFrEF. — Endpoints
- Breye Therapeutics raised a €67.5M (~$76.5M) Series A co-led by Mission BioCapital and Novo Holdings, joined by Sound Bioventures, EIFO, Catalyze Capital and Pappas Capital, taking oral danegaptide into a 2027 Phase 2 in NPDR. — GlobeNewswire
- Aptadir Therapeutics raised a €40M ($45M) seed led by 4BIO Capital, joined by EXTEND (CDP Venture Capital, Angelini Ventures, Evotec), Indaco Venture Partners, XGEN Venture and CE-Ventures, to advance DNA-demethylating RNA lead CAP1-FMR1 in Fragile X syndrome. — GlobeNewswire
IPOs / Follow-Ons
- Immix Biopharma (IMMX) priced a $125M offering, 11.36M shares at $11.00 (J.P. Morgan sole bookrunner), to fund NXC-201 in relapsed/refractory AL amyloidosis. — BioSpace (PR)
- Pyxis Oncology (PYXS) priced ~$110M at $2.90 per share with warrants at $3.50 (up to $282.6M if exercised; Leerink/Guggenheim/Wells Fargo) to take ADC micvotabart pelidotin into a Phase 3 in 2L+ head and neck cancer. — GlobeNewswire
Academic Corner - A phase 1 WRN inhibitor study in microsatellite instability cancers pointed to a new synthetic lethal approach beyond checkpoint blockade. — Nature Medicine
- Oral GLP-1 safiglipron reduced HbA1c by up to 1.45% versus placebo over 32 weeks in early type 2 diabetes. — Nature Medicine
- A phase 1 trial of RO7589831 in MSI and mismatch repair-deficient tumors reported partial responses without reaching maximum tolerated dose. — Nature Medicine
- Nature RDD surveyed the cellular landscape of druggable RNA-binding proteins and emerging targeting strategies. — Nature RDD
- Researchers used integrative DEER modeling to reconstruct hidden GPCR conformations and ligand binding modes. — Structure
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