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Monday, August 25, 2025

Intellia Therapeutics (NTLA) — Investor Brief (Aug 2025) - A leader in Crispr/Gene editing advancements!

 


As NTLA's phase 3 trials continue to show signs of real breakthroughs, there may eventually be takeover offers. (Speculation)

Full disclosure: We are long NTLA!

Intellia Therapeutics (NTLA) — Investor Brief (Aug 2025)

What They Do

  • Leader in CRISPR gene editing.

  • Focused on one-time treatments for serious diseases, using lipid nanoparticles (LNPs) to deliver CRISPR directly into the body.

  • First company to show human proof that CRISPR can work safely in vivo (inside the body).


Key Drugs in Development

  1. nex-z (ATTR Amyloidosis)

    • Targets a deadly heart and nerve disease.

    • In Phase 3 trials now.

    • Competes with Pfizer (tafamidis), BridgeBio (acoramidis), and Alnylam (Amvuttra).

    • Big advantage if proven: a single treatment vs. lifelong pills/injections.

  2. lonvo-z (HAE – Hereditary Angioedema)

    • Rare, painful swelling disorder.

    • In Phase 3 trials, with FDA filing expected in 2026.

    • Current drugs (Takeda’s Takhzyro, CSL’s Andembry) require regular injections. Intellia aims for a one-time permanent fix.


Partnerships

  • Regeneron: Co-develops ATTR drug, but keeps 25% of profits.

  • Other smaller partnerships (Novartis, AvenCell) give extra validation.


Financial Snapshot (Q2 2025)

  • Cash: $630M (enough into early 2027).

  • Quarterly revenue: $14M (partnerships).

  • Quarterly loss: $101M (normal for biotech).

  • Shares: ~105M.


Why It Matters

  • ATTR market: Pfizer’s tafamidis alone made $5.4B in 2024.

  • HAE market: Already over $1B+ yearly and growing.

  • If Intellia succeeds, these are multi-billion dollar markets with limited competition for one-time cures.


Potential Buyers

  • Regeneron: Most likely buyer (already partner, would keep all ATTR profits).

  • Takeda: HAE leader; may want to defend franchise.

  • CSL: Just launched a new HAE drug but could be disrupted.

  • Pfizer: ATTR giant, could buy to protect tafamidis sales.

  • Other big pharma (AstraZeneca, Roche, GSK) could also step in.


Buyout Scenarios

  • Current stock price: ~$11–12/share.

  • If late-stage trials succeed, potential takeout offers could land $28–$57/share (150%–400% upside).

  • Upside comes from:

    • Clear Phase 3 wins.

    • Big Pharma wanting to protect/expand rare disease franchises.

    • Intellia being one of the few with proven in-body CRISPR results.


Risks

  • Competition is fierce: ATTR already has 3 approved drugs.

  • Payers/insurance: One-time therapy pricing will face tough negotiations.

  • Cash burn: Enough until 2027, but commercialization will need more funding.

  • Profit share: ATTR economics partly belong to Regeneron.


Bottom Line

Intellia sits at the intersection of category-defining technology and late-stage assets in two sizable rare-disease markets. With clear Phase 3 wins, especially in HAE and ATTR-CM, we think (i) Regeneron is the most logical acquirer (economics synergy), with Takeda and CSL plausible strategic bidders given franchise logic; and (ii) a reasonable buyout range would center around $28–$57/share, skewing higher if both programs hit convincingly and a strategic (vs. financial) buyer leads the process.



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