Pharma Phriday (March 27, 2026)

Written by Duncan Emerton, Pharma Phriday is a comprehensive weekly update on the pharmaceutical industry, covering clinical trial results, regulatory approvals, corporate development activity, and other significant developments. In this week's note:

Clinical:

• BrightGene announces positive Phase I results for BGM0504 in obesity

• Dizal announces positive results for sunvozertinib in 1L NSCLC

• Novo Nordisk’s Triple G delivers Phase II hit in Chinese diabetes study

• Pfizer’s Lyme disease vaccine heads to FDA despite primary endpoint miss

• Sarepta announces first data from siRNA pipeline targeting FSHD1 and DM1

Regulatory:

• BMS receives US and EU approval for Opdivo in classical HL

• Chugai’s Lunsumio/Polivy combo approved for RR-LBCL in Japan

• Denali’s Avlayah gets FDA nod for Hunter Syndrome

• FDA approves Novo Nordisk’s Wegovy HD

• FDA dances to Rhythm’s tune for second Imcivree approval

• GSK’s risvutatug rezetecan granted Orphan Drug Designation for SCLC in Japan

Commercial:

• Biogen licenses subQ biologics formulation technology from Alteogen

• Gilead to acquire Ouro Medicines for $2 billion

• Kali Therapeutics buddies up with Sanofi on T‑cell engager R&D

• Merck & Co. to acquire Terns Pharma for $6.7 billion

• Novartis acquires pan‑mutant‑selective PI3Kα inhibitor

• Novartis agrees to acquire Excellergy for $2 billion

• Quotient Therapeutics collaborates with Merck & Co. on IBD R&D

Everything Else:

• Aardvark halts trials for obesity candidate, ARD‑201

• Brightseed launches clinically validated, enterprise AI platform

• Earendil Labs announces $787 million in new financing

• Insmed lines up label expansion for Arikayce in MAC lung disease

• Ionis’s zilganersen NDA for Alexander disease accepted by FDA

• Lexicon/Novo Nordisk begins Phase I trial for LX9851 in obesity

• New therapy provides hope for spina bifida

• Ocugen’s OCU410 storms to Phase II ArMaDa trial win

• Oryon Cell Therapies reports Phase Ib/IIa data for Parkinson’s cell therapy

• Roche halts development of emugrobart in SMA and FSHD

• Viz.ai and Alnylam collaborate to improve cardiac amyloidosis detection and care

Clinical

BrightGene announces positive Phase I results for BGM0504 in obesity

What happened. BrightGene has announced positive topline results from Phase I clinical studies of BGM0504, an investigational oral GLP-1/GIP dual receptor agonist, in adult participants in China and the US, including healthy volunteers and individuals with obesity. Across both studies, BGM0504 was generally well tolerated, with no serious adverse events. Preliminary efficacy results based on an ITT analysis showed meaningful reductions in body weight, with mean body weight reductions ranging from 1.0% to 5.6% after four weeks of treatment in the China study (n=75) and from 2.7% to 8.2% after five to eight weeks of treatment in the US study (n=80).

Why this matters. These additional data points confirm the potential of BGM0504 as a weight management strategy in patient with obesity, adding to the already announced data where ≥20% reductions were achieved in the 10mg and 15mg groups of a Chinese Phase II study. It’s too early to compare BGM0504 to other oral GLP-1/GIP dual receptor agonists (e.g., Regeneron’s olatorepatide, Roche’s CT-388, Hengrui’s HRS9531 and Viking’s VK-2735), but with mid- to late-stage trials underway, more data is expected in the next 12-18 months.

Dizal announces positive results for sunvozertinib in 1L NSCLC

What happened. Dizal has announced that its multinational Phase III WU-KONG28 study evaluating Zegfrovy (sunvozertinib) monotherapy as 1L treatment in NSCLC with EGFR exon 20 insertion mutations (exon20ins) met its primary endpoint with positive topline results. The findings suggest that sunvozertinib monotherapy has the potential to become the first and only chemo free, oral agent to treat newly diagnosed NSCLC patients with EGFR exon20ins. WU-KONG28 is a multinational, open-label, randomized confirmatory Phase III study evaluating sunvozertinib versus platinum-based chemotherapy as first-line treatment in advanced NSCLC patients with EGFR exon20ins. The primary endpoint is PFS assessed by blinded independent central review (BICR). Topline results demonstrated that sunvozertinib significantly improved PFS compared to platinum-based doublet chemotherapy, with meaningful clinical benefit. Detailed data from the primary analysis will be submitted for presentation at an upcoming international scientific congress. Sunvozertinib was previously approved in both China and the US for the treatment of RR/NSCLC with EGFR exon20ins. In the 1L setting, sunvozertinib has been granted Breakthrough Therapy Designations by both the FDA and China’s CDE.

Why this matters. The current 1L treatment for NSCLC patients with EGFR Ex20ins is Rybrevant (amivantamab, J&J). Recall, Rybrevant was originally approved in May 2021 as a 2L treatment for NSCLC patients with EGFR Ex20ins, with Rybrevant in combo with chemotherapy being approved as a 1L treatment in March 2024. A SC formulation, Rybrevant Faspro, was approved by the FDA in February 2026. These new data for sunvozertinib now raise the bar for other oral, irreversible TKI’s including firmonertinib (ArriVent), enozertinib (ORIC), and zipalertinib (Taiho/Cullinan) in 1L EGFR Ex20Ins+ NSCLC.

Novo Nordisk’s Triple G delivers Phase II hit in Chinese diabetes study

What happened. Novo Nordisk and United Biotechnology have announced topline results from a Chinese Phase II trial of UBT251, a triple agonist of the receptors for GLP-1, GIP, and glucagon (Triple G), in patients with diabetes. Recall, Novo Nordisk licensed rights to UBT251 from United Biotechnology in March 2025. The trial investigated the safety and efficacy of once-weekly injectable 2mg, 4mg and 6mg doses of UBT251 compared to placebo and semaglutide 1mg in Chinese people with type 2 diabetes. From a baseline mean HbA1c of 8.12%, the highest mean HbA1c reduction observed for people treated with UBT251 was 2.16% compared to 1.77% for the semaglutide 1mg group and 0.66% for the placebo group after 24 weeks of treatment. From a baseline mean body weight of 80.1 kg and a mean BMI of 29.1 kg/m², the mean body weight reduction in the UBT251 groups was up to 9.8% compared with 4.8% in the semaglutide 1mg group and 1.4% in the placebo group. Moreover, UBT251 showed improvements relative to placebo on key secondary endpoints, including waist circumference, blood pressure and lipids. The safety and tolerability profile of UBT251 appeared consistent with what has been observed in other clinical trials with triple-G agonists.

Why this matters. Follows promising Phase II data for UBT251 in obesity announced in February 2026, where the highest mean weight loss observed for people treated with UBT251 was 19.7% (-17.5 kg) compared to 2.0% (-1.6kg) in the placebo group after 24 weeks of treatment. The only caveat is that global proof-of-concept data are two years away, so while promising data have been seen in obesity and diabetes, it’s still early days. Other triple Gs to watch out for include efocipegtrutide (Hanmi) and retatrutide (Eli Lilly), which delivered positive Phase III data in March 2026.

Pfizer’s Lyme disease vaccine heads to FDA despite primary endpoint miss

What happened. Pfizer and Valneva have announced topline results from the Phase III VALOR trial of its investigational 6-valent OspA-based Lyme disease vaccine candidate PF-07307405, demonstrating vaccine efficacy of 73-75% in two pre-specified analyses (i.e., measuring the rate of reduction in confirmed Lyme disease from either Day 28 post last dose or from Day 1 post last dose). Pfizer noted that due to fewer than anticipated Lyme disease cases being accrued over the study period, the pre-determined statistical criterion (95% confidence interval lower bound >20) was not met in the first pre-specified analysis (primary endpoint). Despite this miss, Pfizer intends to submit the vaccine to the FDA for review.

Why this matters. This result, and Pfizer’s intention to submit PF-07307405 to the FDA for review, does introduce risk to the filing, but overall the data are in line with other prophylactic vaccines (e.g., flu vaccine efficacy can be ~40-60%). Moreover, there is unmet need here with no vaccines approved, so the approval bar is already in line with what Pfizer has delivered. Recall, Lyme disease has been a policy focus area for HHS Secretary, RFK Jr.

Sarepta announces first data from siRNA pipeline targeting FSHD1 and DM1

What happened. Sarepta Therapeutics has shared the first clinical results from two of its siRNA programs for neuromuscular diseases; SRP-1001 for facioscapulohumeral muscular dystrophy type 1 (FSHD1) and SRP-1003 for myotonic dystrophy type 1 (DM1). Recall, Sarepta gained access to these programmes via its $500 million upfront licensing and collaboration deal with Arrowhead Pharmaceuticals in November 2024. Early results from Phase I/II ascending dose studies of SRP-1001 for FSHD1 and SRP-1003 for DM1 demonstrated dose- dependent muscle exposure, early biomarker effects, and favorable tolerability, reinforcing scientific confidence in the potential for differentiated benefits of the αvβ6 integrin-targeted delivery platform. In addition, Sarepta has generated proof-of-concept data which found that after a single dose, both SRP-1001 and SRP-1003 support reduction, or knockdown, of the target protein or mRNA. In both studies, most adverse events were mild to moderate and were not dose dependent.

Why this matters. While far from definitive, this validates Sarepta’s bet on RNA interference as a potentially more controllable and repeatable modality versus one-time gene therapies. The timing is also critical. Following safety controversies around Elevidys and pipeline setbacks, the siRNA platform offers both scientific diversification and reputational repair. The company has already prioritised these assets as “high impact” programmes within a broader restructuring. Competitively, Sarepta is entering an increasingly crowded RNA therapeutics landscape. Avidity Biosciences, recently acquired by Novartis, is advancing antibody-oligonucleotide conjugates in similar neuromuscular indications. Other deals include Novartis (licensing deal with Argo Biopharmaceutical, licensing deal with Arrowhead), Madrigal Pharmaceuticals (licensing agreement with Suzhou Ribo Life Science) and Genentech (siRNA R&D collaboration with SanegeneBio).

Regulatory

BMS receives US and EU approval for Opdivo in classical HL

What happened. Bristol Myers Squibb (BMS) has announced that Opdivo (nivolumab) has received approval for two new classical Hodgkin Lymphoma (cHL) indications in the US and the EU. The FDA granted approval of Opdivo in combination with doxorubicin, vinblastine and dacarbazine (AVD) for the treatment of adult and paediatric patients 12 years and older with previously untreated, Stage III or IV cHL. In the EU, the EC approved Opdivo in combination with brentuximab vedotin for the 2L treatment of children 5 years of age and older, adolescents, and adults up to 30 years of age with relapsed or refractory cHL. The US approval is based on the Phase III SWOG 1826 study, evaluating Opdivo in combination with AVD for adult and paediatric (12 years and older) patients with previously untreated Stage III or IV cHL. A submission based on SWOG 1826 study is also currently under evaluation by the EMA. The EU approval is based on the Phase II CheckMate-744 study, evaluating Opdivo in combination with brentuximab vedotin for the 2L treatment of children 5 years of age and older, adolescents and adults up to 30 years of age with relapsed or refractory cHL.

Why this matters. The US and European treatment strategies for classical Hodgkin lymphoma (cHL) are still built on the same backbone, but the US is moving faster toward immunotherapy-based frontline regimens, while Europe remains more conservative and chemotherapy-centric. A key driver of this divergence is the treatment guidelines. The US follows the NCCN guidelines, which call for faster adoption of novel agents (e.g., checkpoint inhibitors, ADCs), whereas in Europe the ESMO guidelines are more conservative, prioritising long-term toxicity data and cost-effectiveness. What these two approvals for Opdivo mean is that checkpoint inhibition has now formally moved from the relapsed setting to frontline standard of care on both sides of the Atlantic. It also signals the beginning of the end for bleomycin, and possible BEACOPP, both of which are associated with significant toxicity, as immunotherapy has become the backbone of treatment.

Chugai’s Lunsumio/Polivy combo approved for RR-LBCL in Japan

What happened. Chugai has announced that Japan’s MHLW has approved the use of Lunsumio (mosunetuzumab; anti-CD20/CD3 bsAb) plus Polivy (polatuzumab vedotin; anti-CD79b ADC) for the treatment of relapsed or refractory large B-cell lymphoma (RR-LBCL). This marks the first approval in the world for the combination therapy of Lunsumio and Polivy for this indication. Approval was based on results from the Phase III SUNMO study evaluating the efficacy and safety of the combination therapy compared to the regimen of rituximab, gemcitabine, and oxaliplatin (R-GemOx; not approved in Japan) in patients with relapsed or refractory large B-cell lymphoma who are not eligible for autologous hematopoietic stem cell transplantation.

Why this matters. This approval strengthens Chugai’s oncology franchise with a differentiated, antibody-based regimen that Roche has described as the first combination of a bsAb and ADC, with potential for outpatient use and avoidance of traditional chemotherapy. For patients, more options are welcome, especially for people who are transplant-ineligible. Treatment choice will increasingly hinge on sequencing, logistics and toxicity management rather than efficacy alone. The appeal of Lunsumio plus Polivy is that it may broaden access to a high-activity regimen without requiring the infrastructure and wait times associated with personalised cell therapy.

Denali’s Avlayah gets FDA nod for Hunter Syndrome

What happened. Denali Therapeutics has received accelerated FDA approval for Avlayah (tividenofusp alfa), a brain penetrating enzyme replacement therapy indicated for the treatment of neurologic manifestations of Hunter syndrome (mucopolysaccharidosis type II, or MPS II) when initiated in presymptomatic or symptomatic paediatric patients weighing at least 5 kg prior to advanced neurologic impairment. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial. The approval of Avlayah was based on the reduction of a key disease biomarker, cerebrospinal fluid heparan sulfate (CSF HS), as a surrogate endpoint reasonably likely to predict clinical benefit in the treatment of neurologic manifestations of Hunter syndrome. In a Phase I/II study, treatment with Avlayah caused a 91% (95% CI: 89%, 92%) reduction in CSF HS from baseline by week 24 of treatment. At week 24, 93% (41 of 44) of Avlayah treated patients had CSF HS levels within the range of individuals without Hunter syndrome. The most common adverse reaction in the study was infusion-related reactions. The ongoing global Phase II/III COMPASS study is designed to generate confirmatory evidence and support global regulatory submissions for Avlayah.

Why this matters. With Avlayah able to cross the blood-brain barrier patients can now be treated for the neurological symptoms of Hunter syndrome, as opposed to just the systemic symptoms (e.g., skeletal/joint issues, upper respiratory tract infections, cardiomyopathy, hypertension, severe hearing loss, etc.). Following Regenxbio’s February 2026 CRL from the FDA for its Hunter Syndrome gene therapy, RGX-121, this is good news for the Hunter Syndrome community. Other developments to follow in the field include a gene therapy being assessed at University College London in the UK, the first patient being dosed in November 2025.

FDA approves Novo Nordisk’s Wegovy HD

What happened. The FDA has approved Novo Nordisk’s Wegovy HD (once-weekly injectable semaglutide 7.2mg) to reduce excess body weight and maintain weight reduction long-term, the first product approved using a Commissioner’s National Priority Voucher (CNPV). The accelerated approval is based on results from the STEP UP trial which showed that semaglutide 7.2mg injected once weekly demonstrated 20.7% and 14.1% mean weight loss in participants with obesity and type 2 diabetes, respectively. In both trials, the safety and tolerability profile of semaglutide was reaffirmed with semaglutide 7.2mg, which was comparable to previous trials with semaglutide for weight management.

Why this matters. While efficacy of semaglutide 7.2mg is still slightly lower than tirzepatide (SURMOUNT-1 showed a 22.5% efficacy estimand on weight loss), approval of Wegovy HD allows Novo to offer a product with a weight loss profile that narrows the gap in efficacy. It also provides Novo Nordisk with an opportunity to slow switching of patients from injectable semaglutide to oral semaglutide and tirzepatide.

FDA dances to Rhythm’s tune for second Imcivree approval

What happened. Rhythm Pharmaceuticals has announced that the FDA has approved an expanded indication for Imcivree (setmelanotide), a first-in-class melanocortin-4 (MC4) receptor agonist, to treat patients living with acquired hypothalamic obesity (HO). Acquired HO is a rare disease characterised by accelerated and sustained weight gain caused by an injury to the hypothalamus or hypothalamic dysfunction. With this label expansion, Imcivree is indicated to reduce excess body weight and maintain reduction long term in adults and paediatric patients aged 4 years and older with acquired HO. The approval is supported by the positive pivotal Phase III TRANSCEND trial of setmelanotide in 142 patients with acquired HO. The global study met its primary endpoint, with a statistically significant -18.4% placebo-adjusted reduction in body mass index (BMI). Imcivree is also approved in the US and Europe in adult and paediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency.

Why this matters. Acquired HO broadens the commercial story for Imcivree into a larger population that the company estimates at around 10,000 people in the US. It also reinforces Rhythm’s core thesis that targeting the MC4R pathway can work outside narrowly defined genetic disease. There are no other approved drugs for acquired HO, but the approval raises the bar for follow-ons in the MC4R field. Palatin has said its oral MC4R agonist PL7737 is being evaluated for hypothalamic obesity, with preclinical data being announced in November 2025. Superluminal Medicines has said its selective MC4R agonist is being advanced with hypothalamic obesity among its intended targets. For patients, Imcivree is far from perfect (due to warnings and common adverse reactions include skin hyperpigmentation, nausea, vomiting and headache), but for a disease with few effective options, the arrival of a mechanism-based therapy is a meaningful shift in the right direction.

GSK’s risvutatug rezetecan granted Orphan Drug Designation for SCLC in Japan

What happened. GSK has announced that risvutatug rezetecan (Ris-Rez, formerly HS-20093), a B7-H3-targeted ADC, has received Orphan Drug Designation (ODD) from Japan’s Ministry of Health, Labour and Welfare for the treatment of small-cell lung cancer (SCLC). The ODD was supported by preliminary clinical data showing durable responses in patients with extensive-stage SCLC (ES-SCLC) who were treated with Ris-Rez in the Phase I ARTEMIS-001 clinical trial.1 This is the sixth regulatory designation for Ris-Rez, which is being developed in a range of solid tumours, including lung, prostate and colorectal cancers. Recall, GSK licensed Ris-Rez from Hansoh Pharma in December 2023.

Why this matters. From GSK’s perspective, this is less about immediate revenue and more about signalling that Ris-Rez is moving from interesting early asset to serious late-stage contender in a very difficult disease. The caveat is that orphan status is a regulatory milestone, not proof that Phase III will work, but it does reinforce momentum around a programme GSK clearly sees as strategically important in solid tumours.

Commercial

Biogen licenses subQ biologics formulation technology from Alteogen

What happened. Alteogen has announced today that it has entered into an exclusive license agreement with Biogen for the development and commercialisation of SC formulations of two biologics utilising ALT-B4 (berahyaluronidase alfa). Alteogen will receive an upfront payment of $20 million and is eligible to receive an additional $10 million upon initiation of development of the second product. In addition, Alteogen is eligible to receive up to $549 million in development, regulatory, and sales milestone payments for the two products combined. Following commercialization, Alteogen will be entitled to receive royalties based on net sales.

Why this matters. An interesting deal that raises some IP related questions, considering that Halozyme (another manufacturer of recombinant human hyaluronidase PH20) has not been slow in seeking to defend its IP against other companies that have licensed Alteogen’s ALT-B4. In April 2025 Halozyme sued Merck & Co. over its SC version of Keytruda, which used Alteogen’s ATL-B4 instead of Halozyme’s rHuPH20. While the case in the US is ongoing, Halozyme has announced that a German court has granted its request for a preliminary injunction ordering Merck & Co. to refrain from distributing and offering Keytruda SC in Germany. Other companies have also used ATL-B4, including AstraZeneca and Daiichi Sankyo.

Gilead to acquire Ouro Medicines for $2 billion

What happened. Gilead Sciences has announced it has entered into a definitive agreement to acquire Ouro Medicines. The acquisition adds OM336 (gamgertamig), a clinical‑stage BCMAxCD3 T cell engager, to Gilead’s inflammation portfolio. OM336 causes rapid and deep B cell depletion following a limited SC administered treatment course. In ongoing Phase I/II clinical studies, OM336 has demonstrated transformative efficacy and a differentiated safety profile after a single treatment cycle in severe antibody-mediated orphan diseases including autoimmune haemolytic anaemia (AIHA) and immune thrombocytopenia (ITP). Gamgertamig has been granted both Fast Track and Orphan Drug Designation by the FDA for the treatment of AIHA and ITP and is expected to enter registrational studies in 2027. Under the terms of the agreement Gilead will acquire all outstanding equity of Ouro Medicines for a total of $1.675 billion upfront and up to $500 million in contingent milestone payments. 

Why this matters. The proposed acquisition of Ouro Medicines follows several other recent deals in which Gilead has either licensed assets or acquired companies as it looks to diversify away from HIV and expand its oncology footprint (e.g., Pregene Biopharma, Sprint Bioscience, Genhouse Bio, Arcellx). While this deal is primarily focused on OM336’s use in I&I indications, there is potential for expansion into multiple myeloma as BCMAxCD3 bispecifics are gaining popularity in the treatment of RRMM. Approved products include Tecvayli (teclistamab; J&J) and Elrexfio (elranatamab; Pfizer), and investigational assets include ABBV-383 and etentamig, both from AbbVie. Interestingly, Gilead is in discussions with Galapagos with respect to a potential research and development collaboration on the acquired Ouro Medicines assets. The parties would split the upfront and milestone payments equally. Galapagos would be solely responsible for development costs through the initiation of registrational studies, after which registrational study costs would be shared equally. One could argue that this forms part of Galapagos’ transformation, a part of which was the winding down of its cell therapy business in October 2025.

Kali Therapeutics buddies up with Sanofi on T-cell engager R&D

What happened. Kali Therapeutics has announced that it has entered into a license agreement with Sanofi for a tri-specific T-cell engager, which has the potential to treat a broad range of B cell-mediated autoimmune diseases. Under the agreement, Sanofi will obtain exclusive worldwide rights to KT501, a novel tri-specific antibody (tsAb) utilising Kali Therapeutics’ proprietary discovery and research platform. KT501 is being evaluated in a Phase I safety, tolerability, PK and PD study in participants with rheumatoid arthritis. Under the terms of the deal, Kali Therapeutics will receive upfront and near-term payments totaling $180 million and will also be eligible to receive up to a total of $1.05 billion in development and commercial milestone payments and tiered royalties on product sales ranging from the high-single to double digits.

Why this matters. Sanofi has an interesting history with TCEs. It previously bought T-cell engager specialist Amunix in February 2022 and later divested three TCE assets to Vir Biotechnology in September 2024. This deal shows it still sees value in the format, albeit in autoimmune disease rather than oncology, a move that fits its broader push to deepen immunology ahead of future competitive pressure on Dupixent. Interestingly, Vir Biotechnology has recently partnered with Astellas on one of the assets acquired from Sanofi, namely SAR446329 (now called VIR-5500), which is dual-masked CD3 T-cell engager (TCE) targeting prostate-specific membrane antigen (PSMA) for the treatment of prostate cancer currently in Phase I trials. Other TCE-focused deals include UCB partnering with Antengene on the development, manufacture and commercialisation of ATG-201, a CD19/CD3 bispecific TCE antibody targeting B cell-related autoimmune diseases.

Merck & Co. to acquire Terns Pharma for $6.7 billion

What happened. Merck & Co. and Terns Pharma have announced that the companies have entered into a definitive agreement under which Merck & Co., through a subsidiary, will acquire Terns Pharmaceuticals $6.7 billion. Terns Pharma’s lead candidate, TERN-701, is a novel investigational oral allosteric BCR::ABL1 TKI currently being evaluated in the Phase I/II CARDINAL trial for patients with Philadelphia chromosome-positive (Ph+), chronic phase chronic myeloid leukaemia (CML) previously treated with at least one prior TKI and who experienced treatment failure, suboptimal response or treatment intolerance. In March 2024, the FDA granted Orphan Drug Designation for TERN-701 for the treatment of CML.

Why this matters. In many ways this deal stands on the shoulders of giants, coming 25 years after the FDA approval of Gleevec (imatinib), the first BCR::ABL1 TKI approved for the treatment of for Ph+ CML. TERN-701 has compelling early data, but it is still early-stage and not yet head-to-head with approved drugs. The obvious benchmark is Novartis’ Scemblix (asciminib), which is approved by the FDA for newly diagnosed Ph+ CML in chronic phase, previously treated Ph+ CML in chronic phase, and T315I-mutant Ph+ CML in chronic phase. Other established BCR::ABL TKIs remain entrenched, including dasatinib (Sprycel) for newly diagnosed and resistant/intolerant Ph+ CML, bosutinib (Bosulif) for newly diagnosed or resistant/intolerant chronic-phase disease, nilotinib (Tasigna) for newly diagnosed chronic-phase disease and imatinib-resistant/intolerant disease, and ponatinib (Iclusig) for selected resistant/intolerant or T315I-positive CML.

Novartis acquires pan-mutant-selective PI3Kα inhibitor

What happened. Novartis has announced that it has entered into an agreement with Synnovation Therapeutics to acquire SNV4818, a pan-mutant‑selective PI3Kα inhibitor, exploring a next-generation approach for the treatment of patients with HR+/HER2- breast cancer and potentially other solid tumour indications. SNV4818 is an oral drug currently being evaluated in a Phase I/II study (NCT06736704) for breast cancer and other advanced solid tumours. Under the terms of the agreement, Novartis will pay $2 billion upfront and up to $1 billion in milestone payments to Synnovation Therapeutics to acquire Pikavation Therapeutics, a wholly owned subsidiary of Synnovation that holds a portfolio of pan-mutant selective PI3Kα inhibitor programs, including SNV4818. The transaction is expected to close in H1 2026, subject to the satisfaction or waiver of customary closing conditions, including regulatory approvals. 

Why this matters. Launched in January 2024 with a $102 million Series A, Synnovation Therapeutics was founded by a team of ex-Incyte veterans to develop targeted small-molecule oncology therapies. The SNV4818 programme aligns strongly with Novartis’s commitment to developing breast cancer treatments, including CDK inhibitors (e.g., Kisqali) and endocrine therapies (e.g., Letrozole). Novartis also markets another PIK3CA inhibitor, Piqray (alpelisib), which hits both the mutant and wild-type forms of PI3Kα. And because inhibition of wild-type PI3Kα is linked to severe hyperglycaemia (plus other side effects like GI disturbance), this creates a significant tolerability problem, limiting how patients can be dosed and how it’s used in combo regimens. SNV4818 is designed to selectively target PI3Kα mutations while sparing wild-type PI3Kα, with the aim of reducing unwanted side effects and improving tolerability. SNV4818 is not alone in the mutant-selective PI3Kα space. Eli Lilly's tersolisib is already in Phase III trials (1L setting in combo with a CDK4/6 inhibitor plus endocrine therapy) and Relay Therapeutics has picked the 2L setting for zovegalisib for its first Phase III trial. OnKure's OKI-219, a mutant-selective PI3Kα inhibitor, is in Phase I trials.

Quotient Therapeutics collaborates with Merck & Co. on IBD R&D

What happened. Quotient Therapeutics has announced that it has entered a multi-year research collaboration agreement with Merck & Co. to discover novel drug targets in inflammatory bowel disease (IBD) using Quotient’s somatic genomics platform technology. Somatic genetic mutations naturally accumulate over a lifetime, resulting in trillions of different genomes within an individual. Through its platform, Quotient interrogates patient tissue for these mutations within the context of disease to find mutations that cause or protect from that disease. These insights can inform novel therapeutic strategies for a broad range of diseases, including IBD. Under the terms of the agreement, Quotient will receive an upfront payment of $20 million. Quotient is also eligible to receive development, regulatory and commercial milestones for a potential total deal value of up to $2.2 billion.

Why this matters. From Quotient’s perspective, this is strong platform validation. Merck & Co. is effectively paying for a new route to human biology, not a finished drug. Form Merck & Co. it complements rather than replaces existing IBD assets. For example, Merck already has tulisokibart (anti-TL1A) in Phase III for ulcerative colitis and Crohn’s disease, so this deal broadens its shot at future mechanisms rather than filling an empty pipeline. For the competition, this just makes the IBD market that little bit more crowded as there are multiple small molecules (e.g., Rinvoq, Zeposia) and biologics (e.g., Skyrizi, Tremfya, Simponi, Entyvio) all FDA approved for at least one major IBD setting, with several now spanning both UC and Crohn’s disease.

Everything Else

Aardvark halts trials for obesity candidate, ARD-201

Aardvark Therapeutics has voluntarily paused development of its obesity candidate, ARD-201. This voluntary pause includes the Phase II POWER trial evaluating a FDC of ARD-201/DPP-4i for the treatment of obesity in the prevention of weight regain among patients who have successfully lost approximately 15% of body weight on GLP-1RA therapy, and the Phase II STRENGTH trial to evaluate placebo-adjusted weight loss and the additive effects of ARD-201 combined with GLP-1RA.

Brightseed Launches clinically validated, enterprise AI platform

Brightseed, an AI-native life sciences company, has announced the launch of its innovation platform, what the company claims is the first continuous, AI-powered health sciences innovation platform designed to increase the probability of commercial success across health and life sciences. With this launch, Brightseed formally evolves from a discovery-focused bioactives company into a platform-based enterprise AI company delivering data-as-a-service (DaaS) to global health science organizations. The business evolution is built on the technology and learnings that has allowed Brightseed to produce the first clinically validated, AI discovered compounds in the commercial market.

Earendil Labs announces $787 million in new financing

Earendil Labs has announced that it has raised $787 million in financing rounds. The funding will accelerate Earendil Labs' AI-driven R&D platform, expand its interdisciplinary teams, and advance a growing pipeline of antibody and biologics programmes, with the goal of delivering first-in-class and best-in-class therapies for patients with serious diseases. The company's AI-native platform has produced more than 40 programmes, including HXN-1001, a half-life extended anti-TL1A antibody which is ready for Phase II clinical development, together with multiple IND submissions planned in 2026 and 2027. Sanofi partnered with Earendil in January 2026 on the development of two bsAbs, HXN-1002 and HXN-1003.

Insmed lines up label expansion for Arikayce in MAC lung disease

Insmed has announced positive topline results from the Phase IIIb ENCORE study. This study evaluated Arikayce (amikacin liposome inhalation suspension) plus multidrug therapy (azithromycin 250mg + ethambutol 15mg/kg) once-daily versus placebo plus multidrug therapy once-daily in diagnosed patients with a new occurrence of Mycobacterium avium complex (MAC) lung infection who had not received antibiotics. The Arikayce regimen showed both statistically significant and clinically meaningful improvements in respiratory symptom score (RSS) and culture conversion rates. 

Ionis’s zilganersen NDA for Alexander disease accepted by FDA

Ionis Pharmaceuticals has announced that the FDA has accepted for Priority Review the New Drug Application (NDA) for zilganersen, an investigational RNA-targeted medicine for Alexander disease, a rare, progressive and often fatal neurological condition. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of September 22, 2026. The NDA and Priority Review designation were based on results from the Phase I-III pivotal study of zilganersen in children and adults living with Alexander disease. In the study, zilganersen 50mg demonstrated statistically significant and clinically meaningful stabilisation on the primary endpoint of gait speed as assessed by the 10 Metre Walk Test (10MWT) compared to control at week 61 with favorable safety and tolerability.

Lexicon/Novo Nordisk begins Phase I trial for LX9851 in obesity

Lexicon Pharmaceuticals and Novo Nordisk have initiated a Phase I study with LX9851, a first-in-class, oral non-incretin development candidate. In March 2025, Lexicon signed an exclusive license agreement with Novo Nordisk for LX9851 in obesity and associated metabolic disorders. Under the terms of the agreement, Novo Nordisk obtained an exclusive, worldwide license to develop, manufacture and commercialise LX9851 in all indications. The Phase I study is investigating the safety, tolerability, PK and PD of single and multiple ascending doses of LX9851 compared to placebo in 96 people with obesity. The trial is expected to be completed in Q1’27. As part of this deal, Lexicon is eligible to receive upfront and near-term milestone payments of up to $75 million. In total, Lexicon would be eligible to get $1 billion in upfront and potential development, regulatory and sales milestone payments.

New therapy provides hope for spina bifida

A new therapy could prevent lifelong disability for thousands of babies each year, offering hope before birth to families facing spina bifida. Spina bifida is a condition where the spine doesn’t close properly during early development. The result can be paralysis, bladder or bowel problems and lifelong disability. For decades, treatment began only after birth. Now, one of the most promising examples is in utero surgery that combines traditional repair techniques with stem cell therapy. This approach was the focus of The CuRe Trial, the results of which were recently reported in The Lancet.

Ocugen’s OCU410 storms to Phase II ArMaDa trial win

Ocugen has announced positive 12 month data from the Phase II ArMaDa clinical trial evaluating OCU410 (AAV5-RORA), its novel modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). Data show that the planned Phase III dose of OCU410 significantly reduced the size of lesions (31%) vs. placebo. In a January 2026 preliminary analysis, the same dose of OCU410 caused a 54% lesion reduction.  Ocugen plans to initiate the OCU410 Phase III registrational trial in Q4’26.

Oryon Cell Therapies reports Phase Ib/IIa data for Parkinson’s cell therapy

Oryon Cell Therapies has announced new clinical and neuroimaging data from an ongoing Phase Ib/IIa study evaluating its autologous dopaminergic neuron replacement therapy for Parkinson’s disease. The study is being conducted in collaboration with expertise across Mass General Brigham led by the Neuroregeneration Institute at McLean Hospital. Study participants received unilateral neuronal implants. The data show improvements in motor function, together with neuroimaging consistent with restoration of dopaminergic signalling in the transplanted brain region. Interim results from the first five participants showed substantial improvements in motor function. A sixth participant had not yet had their first post-implant assessment. In each of the first five participants, OFF-state MDS-UPDRS Part III motor scores decreased by approximately 29%–62% from baseline at assessments 6–18 months after treatment. Improvements were observed in core Parkinson’s motor symptoms, including bradykinesia, rigidity, and gait.

Roche halts development of emugrobart in SMA and FSHD

Roche has halted development of its anti-myostatin mAb, emugrobart, in spinal muscular atrophy (SMA) and facioscapulohumeral muscular dystrophy (FSHD). The company announced this decision in two separate letters to the SMA and FSHD communities, which both noted that emugrobart failed to demonstrate consistent improvements in muscular growth in their respective mid-stage studies. This decision was based on the outcomes of the Phase II MANOEUVRE trial in FSHD and the Phase II/III MANATEE study in SMA. Development in other indications, particularly obesity, is now being called into question.

Viz.ai and Alnylam collaborate to improve cardiac amyloidosis detection and care

Viz.ai has announced a partnership with Alnylam Pharmaceuticals to accelerate early identification and standardise diagnostic evaluation of patients with cardiac amyloidosis, a serious and underdiagnosed cause of heart failure. Cardiac amyloidosis is a progressive condition in which misfolded proteins deposit in the heart muscle, leading to heart failure and poor outcomes if left untreated. There are two primary types of cardiac amyloidosis: the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) and light chain amyloidosis (AL).