Last updated: September 8, 2026
Novartis’ pelacarsen missed the primary endpoint of the 8,323 patient Lp(a)HORIZON outcomes trial, Novo Nordisk stopped two Phase 3 ziltivekimab heart failure trials early, and semaglutide moved 40.4% of children below the obesity threshold in the STEP Young Phase 3.
This page covers the news from Friday, September 4 through Monday, September 7, 2026: the pelacarsen and ziltivekimab outcomes failures, the STEP Young pediatric obesity data, the Zanvastro approval for Alexander disease, the Menarini and Gan & Lee licensing deal, Boston Scientific’s cyberattack recovery and Infinion CX recall, and the ADARx IPO filing. US markets were closed Monday for Labor Day.
What happened in the Lp(a)HORIZON trial of pelacarsen?
Lp(a)HORIZON, an 8,323 patient Phase 3 outcomes trial of pelacarsen in patients with elevated lipoprotein(a) and established cardiovascular disease, did not meet its primary endpoint. Novartis announced the topline result on September 4, 2026 and disclosed no effect sizes. Data will go to a future medical congress.
Pelacarsen is an antisense oligonucleotide discovered by Ionis Pharmaceuticals and licensed to Novartis. The trial (NCT04023552) tested whether lowering Lp(a) reduces a composite of cardiovascular death, non fatal myocardial infarction, non fatal stroke, and urgent coronary revascularization requiring hospitalization, evaluated in the overall population with Lp(a) of at least 70 mg/dL and in a subpopulation of at least 90 mg/dL. Shreeram Aradhye, Novartis’ President of Development and Chief Medical Officer, said lower Lp(a) levels were observed with pelacarsen but the findings “did not demonstrate that this translated into reduced cardiovascular risk in the overall study population.” No hazard ratio, p value, or Lp(a) reduction percentage was published. Novartis’ US listed shares closed down 1.9% Friday at $159.99; market data, no cause assigned. Royalty Pharma, which holds a royalty interest in pelacarsen, issued a same day update and closed roughly flat.
Why does the pelacarsen failure matter beyond Novartis?
Lipoprotein(a) has the strongest genetic case of any unvalidated cardiovascular target: levels are largely inherited, and decades of genetic epidemiology tie high Lp(a) to heart attack and stroke risk. Lp(a)HORIZON was the first large outcomes test of the hypothesis that lowering it with a drug reduces events. The answer, at least for this drug in this population, was no.
The result lands directly on the programs still running. Amgen’s olpasiran, an siRNA that produces deeper Lp(a) lowering than pelacarsen in earlier studies, remains in the OCEAN(a) Outcomes trial (NCT05581303), which was listed as ongoing as of our September 7 check. Eli Lilly’s lepodisiran, also an siRNA, is in the ACCLAIM outcomes program, and Silence Therapeutics is developing zerlasiran. None of these trials has read out. Their sponsors can still argue that deeper or more durable lowering changes the answer, but the free ride from genetics ended on Friday: the burden of proof now sits on magnitude, not mechanism. The congress presentation of the Lp(a)HORIZON data becomes a class event, because how close the result came to null will define how much room the deeper lowering argument has left.
What did Novo Nordisk stop in the ziltivekimab program?
Novo Nordisk discontinued the HERMES and ATHENA Phase 3 heart failure trials of ziltivekimab, its monthly IL-6 inhibitor, after the data monitoring committee reviewed the totality of the data and concluded there was low likelihood of a different outcome from ZEUS. Only the ARTEMIS trial in patients after an acute heart attack continues, with a readout expected in the first half of 2027.
The discontinuations were reported September 7 by Fierce Biotech, STAT, Endpoints News, Reuters, and Bloomberg, with Novo confirming to reporters; we found no company press release as of Monday night, so the details rest on reporting plus company statements. Fierce Biotech puts HERMES at about 4,900 patients with heart failure with mildly reduced or preserved ejection fraction and ATHENA at about 673 patients in a similar population, with the trials halted early on Friday, September 4. ZEUS, the 6,300 plus patient outcomes trial in atherosclerotic cardiovascular disease, chronic kidney disease, and elevated inflammation, missed its primary endpoint; Novo disclosed the miss in July and stated it plainly in its August 4 second quarter report. Analysts had estimated peak ziltivekimab sales near $3 billion before the failures, per Fierce; treat that as an analyst figure, not a company one.
Taken together with pelacarsen, this was a brutal four days for cardiovascular surrogate biology: an Lp(a) lowering drug and an IL-6 lowering drug both did what they were designed to do to their biomarkers, and neither program survived contact with outcomes data intact. The contrast is Amgen’s Repatha, which reduced death risk 20% in high risk primary prevention in the VESALIUS-CV trial reported August 31: LDL is a surrogate that outcomes trials have validated repeatedly, and the market treats it accordingly. Our coverage of that readout is here.
What did the STEP Young trial show in children with obesity?
In STEP Young, 40.4% of children ages 6 to under 12 treated with semaglutide finished week 68 with a BMI below the obesity threshold, versus 0% of children on placebo. Novo Nordisk announced the topline September 7; full results are slated for ObesityWeek, November 14 to 17, in Washington DC.
The trial enrolled 165 children, more than 85% of whom had severe (class II or III) obesity at baseline. Both arms received a reduced calorie diet and increased physical activity; semaglutide was dosed once weekly at a weight based maximum of 1.7 mg or 2.4 mg. The primary endpoint of superior BMI reduction at week 68 was met, and improvement in BMI classification was a confirmatory secondary endpoint. Novo said safety and tolerability were consistent with prior pediatric and adult semaglutide trials, with no new safety concerns and no concerns for growth or pubertal development. The study runs to week 104 for supportive endpoints and was conducted as part of semaglutide’s post marketing pediatric requirements. For the wider obesity pipeline context, see our triple agonist explainer.
What is Zanvastro and why does its approval matter?
Zanvastro (zilganersen) is Ionis’ antisense oligonucleotide for Alexander disease, a progressive and ultimately fatal leukodystrophy. On September 3 the FDA granted it standard approval as the first and only disease modifying treatment for the disease, in patients 18 months and older, dosed 50 mg quarterly by intrathecal injection.
The approval rests on a 54 participant pivotal trial run at 13 sites in 8 countries, with participants ranging from 1.5 to 53 years old. The primary endpoint, percent change in gait speed on the 10 Meter Walk Test at week 61, showed a 33.3% least squares mean difference (p=0.041) in patients five and older. It is a standard approval, not accelerated, which is notable on a dataset this small, and it came with a rare pediatric disease priority review voucher. Ionis plans a US launch within weeks and has not disclosed a list price; Recordati holds ex US commercialization rights, with European and Japanese submissions expected in 2027.
The same company absorbed both ends of the week: a 54 patient full approval on September 3 and an 8,323 patient partnered outcomes failure on September 4. Ionis shares closed the week essentially flat at $58.09, down 0.1% Friday, which is what a portfolio pricing both events at once looks like. Market data, no cause assigned.
What are the terms of the Menarini and Gan & Lee bofanglutide deal?
Menarini licensed European rights to bofanglutide (GZR18), Gan & Lee’s long acting GLP-1 receptor agonist dosed once every two weeks, for EUR 62 million upfront and up to EUR 664 million in milestones, a total of up to EUR 726 million excluding royalties, plus separate double digit royalties on net sales. The deal was announced September 3 and covers 39 European countries.
Bofanglutide has met primary endpoints in Phase 3 trials in China and Phase 2 trials in the US, and its once every two weeks subcutaneous schedule would halve the injection frequency of weekly incumbents. The construction is standard, with royalties excluded from the headline total, which makes it eligible for our deal ledger as entry #14. Some coverage quotes the deal as $771 million; that is a dollar conversion of the euro total.
How do ledger entries #13 and #14 compare?
Both of the last two Asia origin licenses priced at an 8.5% cash share, meaning the upfront equals 8.5% of the disclosed total, but they sit at opposite ends of the development curve. The table below uses each deal’s own disclosed construction; both exclude royalties from the total. The full ledger method is in our binary ledger reference.
| Entry | Deal (date) | Asset and stage | Upfront | Total (ex royalties) | Cash share |
|---|---|---|---|---|---|
| #13 | GSK / Hutchmed (Sept 3) | HMPL-A830, antibody targeted therapy conjugate, preclinical | $110M | Up to $1.295B | 8.5% |
| #14 | Menarini / Gan & Lee (Sept 3) | Bofanglutide, GLP-1 receptor agonist, Phase 3 met primaries in China | EUR 62M | Up to EUR 726M | 8.5% |
A preclinical oncology asset and a Phase 3 proven metabolic asset clearing at the same cash share suggests the share is measuring something other than stage risk. In both deals the seller kept the piece the buyer valued most: Hutchmed keeps Greater China and leads the Phase 1, Gan & Lee keeps the world outside 39 European countries plus double digit royalties. Our coverage of entry #13 is here.
Where does Boston Scientific’s cyberattack recovery stand?
Boston Scientific said September 4 that shipping has begun for the majority of products at major global distribution centers and that customers can submit orders electronically again, roughly one week after the cyberattack halted shipments. There is still no timeline for full restoration and no quantified financial impact.
In the September 4 update, reported by MedTech Dive from the company’s incident page, Boston Scientific expressed “growing confidence that the unauthorized access was limited to select internal facing IT infrastructure,” said the order backlog will take time to clear, and declined to address manufacturing status. The company is working with CrowdStrike and other outside cybersecurity experts. The week of September 1 target for beginning partial shipping restoration, which had passed unconfirmed, was met one week late. No 8-K quantifying the impact had appeared as of our Monday night check. Boston Scientific shares closed Friday at $47.80, up 1.8%; market data, no cause assigned. The start of this story is covered in our August 28 edition.
What is the Infinion CX spinal cord stimulator recall?
The FDA published an alert September 3 on Boston Scientific’s removal of Infinion CX spinal cord stimulator lead kits, models SC-2317-50 and SC-2317-70, after 1,081 reports of serious injury and no deaths as of May 27, 2026. The agency classifies it as the most serious type of recall.
The leads can experience mechanical stress at the anchor site, which may produce high impedance measurements or lead fractures, causing inadequate stimulation and in some cases surgery to replace the lead. Boston Scientific began the removal with a June 17 customer letter instructing facilities to stop using and return unused affected units; the FDA says patients with implanted leads need routine clinical follow up only. The recall is unrelated to the cyberattack, but the two stories now share a ticker and a month.
What happened at ERS with Generate Biomedicines’ GB-0895?
Generate Biomedicines’ Phase 1 data for GB-0895, its AI engineered long acting anti TSLP antibody, were presented at the European Respiratory Society congress on September 7, the day the embargo lifted. The findings had effectively been public since August 25, when ERS inadvertently posted draft posters about two weeks early.
Per the posters as reported, a single dose in a 40 patient COPD study produced reductions across four COPD biomarkers with a half life near 100 days, and in patients with elevated eosinophils showed broad anti inflammatory activity lasting at least six months; most adverse events were mild or moderate, with six serious events among five patients, none grade 3 or higher. Asthma follow up data showed a 300 mg single dose sustaining activity for at least six months, supporting the twice yearly dosing the company is carrying into its global Phase 3 SOLAIRIA program, which is enrolling about 1,600 patients on a 300 mg every six months schedule. The TSLP pathway already has outcomes validation in severe asthma through AstraZeneca and Amgen’s Tezspire; GB-0895’s bet is that a six month dosing interval on the same target is worth the wait.
What moved in biotech capital markets?
ADARx Pharmaceuticals filed an S-1 for a proposed Nasdaq IPO on September 4, with Renaissance Capital and Bloomberg reporting a $100 million placeholder. The AbbVie allied company has three clinical stage siRNA programs across hepatic and extrahepatic targets.
The filing extends a reopening IPO window we have tracked since the Sentivera launch in August, alongside a 1,600% third quarter rise in biopharma reverse mergers reported by BioSpace. Truist told clients September 4 that “biotech is back,” with first half progress smoothing the glide path through year end, per BioSpace. Separately, Fierce Biotech’s fundraising tracker logged an $80 million raise for NeuShen on September 7. Our IPO window coverage began with the Sentivera piece.
What did not happen this weekend?
Several tracked items produced nothing, and the absences are part of the record. Scope: our searches and index checks the night of Monday, September 7.
Amylyx has still not announced the closing of its $500.2 million offering priced August 19; Friday’s $34.10 close was the sixth consecutive close below the $35.50 offer price, and the greenshoe window forces disclosure by roughly September 18. Lilly’s purchase of Sangamo’s platform assets, expected to close September 4, has no closing announcement. There is still no primary document and no reported formal FDA hold in the Novartis and Bristol Myers Squibb autoimmune CAR T pauses, covered in our September 2 edition. No Senate hearing has been scheduled for FDA commissioner nominee Heidi Overton. Summit and Akeso have not named the congress for the HARMONi-2 overall survival presentation.
Frequently asked questions
Did pelacarsen fail to lower Lp(a)?
No. Novartis said Lp(a) lowering was observed. The trial failed because the lowering did not translate into fewer cardiovascular events in the overall study population. The drug did its biochemical job; the hypothesis did not deliver the clinical payoff.
Is the Lp(a) hypothesis dead?
Not yet, but it is wounded. Pelacarsen is one drug at one level of Lp(a) lowering. Amgen’s olpasiran and Lilly’s lepodisiran produce deeper lowering and their outcomes trials continue. If those also miss, the hypothesis itself is finished; if they win, the field will conclude the lowering threshold matters.
How much did pelacarsen lower Lp(a) in the trial?
Novartis has not said. No reduction percentage, hazard ratio, or p value was disclosed in the September 4 release. The data are promised to a future medical congress.
What is ziltivekimab and what is left of its program?
Ziltivekimab is Novo Nordisk’s monthly anti IL-6 antibody, designed to cut cardiovascular risk by lowering inflammation. After the ZEUS trial missed and the HERMES and ATHENA heart failure trials were stopped early, only ARTEMIS, in patients after an acute heart attack, is still running, with a readout expected in the first half of 2027 per Fierce Biotech.
Why did Novo stop HERMES and ATHENA early?
Per Novo’s statements to media, the data monitoring committee assessed the totality of the data and concluded there was low likelihood of a different outcome from ZEUS. Stopping early spares patients and money on a question the committee considered effectively answered.
What did STEP Young show, in one line?
In 165 children ages 6 to under 12 with obesity, 40.4% on weekly semaglutide reached a BMI below the obesity threshold at week 68, versus 0% on placebo, with safety consistent with prior trials.
Is semaglutide approved for children under 12?
No. STEP Young was conducted under semaglutide’s post marketing pediatric requirements. Any label change would follow regulatory submissions; Novo has not announced timing.
What is Alexander disease?
Alexander disease is an ultra rare, progressive leukodystrophy caused by GFAP mutations, typically appearing in infancy or childhood and ultimately fatal. Zanvastro is the first approved disease modifying treatment.
Why is a 54 patient standard approval notable?
Standard approval requires substantial evidence of clinical benefit, not a surrogate promise to be confirmed later. Granting it on a 54 participant trial with a p value of 0.041 signals FDA flexibility on ultra rare diseases when the endpoint is functional and the natural history is relentless decline.
What is bofanglutide and how is it different from Wegovy or Zepbound?
Bofanglutide (GZR18) is a long acting GLP-1 receptor agonist from Gan & Lee dosed once every two weeks, versus weekly for semaglutide and tirzepatide. It has met primary endpoints in Chinese Phase 3 and US Phase 2 trials; Menarini now holds rights in 39 European countries.
Do I need to do anything if I have an Infinion CX lead implanted?
Per the FDA alert, patients with implanted leads need routine clinical follow up only; additional monitoring is not recommended. The removal applies to unused units in the field. Talk to your physician about any change in stimulation.
Is the Boston Scientific recall related to the cyberattack?
No. The recall stems from mechanical lead failures reported over years, with the customer letter dated June 17. The cyberattack began in late August. They are separate events at the same company.
When will the Lp(a)HORIZON full data be presented?
Novartis has said only that findings will be presented at an upcoming medical congress. No venue or date has been named.
What are the next hard dates to watch in September 2026?
Amylyx must disclose its offering close by roughly September 18, Ultragenyx’s UX111 PDUFA date is September 19, Grail’s Galleri advisory committee meets September 23, AbbVie presents full CERVINO data September 25, Section 232 pharmaceutical tariffs take effect September 29, and Scholar Rock’s apitegromab action date is September 30.
Sources
Primary sources: Novartis media release on Lp(a)HORIZON topline results (September 4, 2026); Novo Nordisk press release on STEP Young Phase 3 data (September 7, 2026); Novo Nordisk Q2 2026 earnings release (August 4, 2026, ZEUS statement); Ionis Pharmaceuticals press release on Zanvastro FDA approval (September 3, 2026); Gan & Lee and Menarini joint release (September 3, 2026); FDA medical device recall alert on the Infinion CX lead (September 3, 2026); Boston Scientific incident page update (September 4, 2026); ADARx Pharmaceuticals Form S-1 (SEC, September 4, 2026); Amgen VESALIUS-CV release (August 31, 2026).
Trade press and market reporting: Fierce Biotech (ziltivekimab discontinuations, September 7; NeuShen fundraising tracker, September 7); STAT, Endpoints News, Reuters, Bloomberg (ziltivekimab, September 7); MedTech Dive (Boston Scientific restoration, September 4; Infinion CX recall coverage); BioSpace (Truist note, September 4); Renaissance Capital (ADARx placeholder); Fierce Pharma (oral GLP-1 tracker, September 4). Market prices are official September 4 exchange closes; US markets were closed September 7 for Labor Day.
