What big pharma is doing in ADPKD

By Dalya Gartzman, Renasis Bio · · Data as of 26 Oct 2026

9 of the 10 largest pharma companies have made a move in ADPKD. 21 of the largest 50.

Including:

Who

Claude and I mapped every ADPKD activity by the 50 largest pharma companies, total 48 activities since 2005:

Figure 1: The top 50 pharma companies by 2024 revenue (Drug Discovery & Development Pharma 50, 2025), with the 21 that have at least one ADPKD activity since 2005 in blue: Merck, Pfizer, J&J, AbbVie, AstraZeneca, Roche, Novartis, Lilly, Sanofi, Novo Nordisk, GSK, Takeda, Boehringer, Bayer, Teva, Vertex, Sandoz, Biogen, Otsuka, UCB and Ipsen. Of the top 10, only Bristol Myers Squibb has none. 48 activities, one marker each: 8 in-house programs, 6 acquisitions, 2 partnerships, 20 venture investments, 10 academic trials, 2 generic tolvaptan launches.
Figure 1. The 21 of the top 50 pharma with an ADPKD activity. Explore them in the pharma map

When

In the past 3 years, 10 pharmas made 13 new ADPKD commitments. More than double the pace of the 10 years before (19 in total).

The involvement changed not only numerically but also categorically. Startup investment leads the surge (7 rounds in 3 years vs. 12 in 10), showing a willingness to take early risk to win here. And AstraZeneca and GSK took new in-house antibodies into the clinic, showing faith in the field's economics and viability.

Figure 2: New ADPKD commitments by the top 50 pharma, by year, 2005 to 2026, one marker per commitment (in-house program, acquisition, partnership, venture investment, academic trial support). 19 in the 10 years 2014 to 2023, about 2 a year; 13 in the 3 years 2024 to 2026, about 4.5 a year, from 10 companies. Each funding round counts once. Excludes generics and acquisitions where ADPKD came along with an asset bought for another disease.
Figure 2. New ADPKD commitments by the top 50 pharma, by year. Explore them in the pharma map

What

🎯 Pharma is moving toward the root cause, just like the VCs in my last post:

Vertex corrects misfolded PC1. Novartis's farabursen blocks miR-17 to boost PC1 and PC2. Biogen-backed Estuary and SonoThera (backed by 5 big pharmas) are developing gene therapies.

Figure 3: ADPKD assets pharma developed, bought or backed. Before 2022, 1 of 10 restores PC1 or PC2: Vertex's PC1 corrector VX-407; the others are tolvaptan (Otsuka), lixivaptan (Roche, investor), everolimus (Novartis), bosutinib (Pfizer), roscovitine and venglustat (Sanofi), Navitor's mTORC1 inhibitor (J&J, GSK, Sanofi, investors), anti-PAPP-A (Calico / AbbVie) and SCO-792 (Scohia, Takeda). 2022 or later, 4 of 9: Renasis Bio's RNA-upregulating small molecules (AstraZeneca, investor), Novartis's farabursen, Estuary's AAV gene therapy for PKD1/PKD2 (Biogen, investor), and likely SonoThera's gene therapy (5 pharmas, investors; target not yet public); the others are AstraZeneca's anti-PAPP-A AZD1613, GSK4771261, Borealis's xRNA platform (Novartis), Mironid's PDE4 activator (Roche, investor) and Anakuria's mTORC1 inhibitor (J&J).
Figure 3. Pharma-backed assets aiming to restore polycystin, before and since 2022. Explore them in the pharma map

How much

💰 The Novartis–Regulus deal is a benchmark: $0.8B upfront plus up to $0.9B more, for an ADPKD asset after Phase 1b. FDA has since agreed to a single pivotal Phase 3.

🚀 Pharma venture arms are increasingly active, too. 11 big pharmas have taken equity in ADPKD startups. In 2026 alone, 6 of them joined rounds totaling $264M.

📊 Adding it up: up to $1.7B for farabursen, ~$480M in startup rounds with pharma investors since 2014, and ~$240M in venture creation (Borealis with Novartis, Scohia with Takeda). Over $2.4B in ADPKD deals and rounds with pharma behind them.

Figure 4: Over $2.4B in ADPKD deals and rounds with pharma behind them: $1.7B for the acquisition of farabursen (Novartis), about $480M in startup rounds with pharma since 2014, and about $240M in venture creation (Borealis with Novartis, Scohia with Takeda). Novartis acquires Regulus, 2025: $0.8B upfront plus up to $0.9B on approval (CVR), for farabursen, an anti-miR-17 oligonucleotide, after Phase 1b. In 2026, 6 pharmas joined $264M in ADPKD rounds: SonoThera Series B $125M (Bayer, Otsuka, UCB, J&J), Estuary Series A $93M (Biogen) and Mironid Series B $46M (Roche). Round totals, not pharma's share.
Figure 4. ADPKD deals and rounds with pharma behind them. Explore them in the pharma map

Why

🇯🇵 Otsuka pioneered the ADPKD market. Tolvaptan was the first ADPKD drug, approved in Japan in 2014 and the US in 2018.

Now tolvaptan is generic: Lupin launched the first US generic in 2025, Apotex, Alkem and Teva followed, and Sandoz has one in Europe.

Otsuka's response? Keep investing in ADPKD. It co-funds combination trials with tolvaptan, and in 2026 it joined SonoThera's Series B. The old guard is funding the new generation.

Figure 5: The old guard is funding the new generation. Timeline of Otsuka's tolvaptan, the only approved ADPKD drug: first trial in 2005, first approval in Japan, FDA approval, then generic. Otsuka reinvests in what comes next: 2022, collaborator on the probenecid plus tolvaptan trial (Mayo Clinic); 2022, design-phase grant for IMPEDE-PKD, a metformin trial; 2024, co-funder of HYDRO-PROTECT, hydrochlorothiazide plus tolvaptan (UMC Groningen); 2026, SonoThera's $125M Series B for gene therapy, alongside Bayer, UCB and J&J.
Figure 5. Otsuka, tolvaptan's maker, funding the next ADPKD therapies. Explore them in the pharma map

To sum up

Pharma's growing involvement in ADPKD signals a Goldilocks moment for the field: validated and lucrative.

For patients, this is great news: pharma can take drugs all the way to them.

For startups like ours, it means exits are real, they come early, and a true pipeline play is within reach: partner the lead asset with pharma, and use that to build the platform.

Sources