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From Rare Disease to Breakthrough? This Under-the-Radar Biotech Could Be Pharma’s Next Big Winner

Author

Noah Zelvis
July 17, 2025

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Mirum Pharmaceuticals, Inc.

July 17 – Pre‑market
Ticker: MIRM | Sector: Biotechnology | Market Cap: ~ $2.6B

30‑Second Take

Why now? This California-headquartered biotech is hot property right now thanks to a compelling combination of rapid revenue growth.

It’s up 60% YoY, driven by strong uptake of its rare liver disease drug, LIVMARLI.

The company has also raised its full-year guidance, is pursuing several international expansion projects, and has multiple late-stage pipeline assets that could unlock new markets in the not-too-distant future.

With bullish analyst sentiment, a strong chart setup, and momentum from recent upgrades, MIRM offers high upside potential. Investors bullish on biotech should keep it firmly on their radar.

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Trade Setup

Time frame: Swing to medium-term
Edge type: Momentum breakout

Snapshot Table

Metric

Value

Current Stance

Price

$52.77

Average

52‑week range

$36.20 - $54.78

Average

Short interest

15.35%

Average

Next catalyst

Q2 earnings, expected August 06

Chart

5-Day Synopsis: After the surge in momentum triggered by FDA approval in Q1, trading has levelled off with prices hovering around the top end of the 52-week range.

The last five sessions have been stable, with no significant gains or losses recorded at the end of any session.

MIRM has accrued a collective loss of 0.94% over the five days, with prices ranging from $51.98 to $54.62.

Bull Case 

Core thesis: Founded in California in 2014, Mirum Pharmaceutical is a clinical biopharmaceutical company dedicated to developing safe and effective therapies for people with rare liver diseases.

It specializes in developing medications that address urgent, unmet needs and fill therapeutic voids.

Its current areas of focus are Cholestasis, Inborn Errors of Bile Acid Metabolism, and Cholesterol Metabolism.

It has developed three FDA-approved medicines: LIVMARLI, Cholbam and CTEXLI™. It maintains clinical trial sites in the United States, Europe, and Australia.

Catalysts: In Q1, Mirum secured FDA approval for an oral tablet formulation of LIVMARLI, which treats liver itch – a condition responsible for up to 82% of liver transplants.

Approval skyrocketed the pharma stock to a 236% year-to-date gain, with further gains expected upon commercial rollout.

Its sales also grew by 61% compared with the prior quarter, to $111.6 million.

Full-year guidance has also been lifted from $435 million to $450 million, reflecting growing sales expectations.

Valuation upside: Analyst price targets span a wide range, with the lowest just $53.00.

The high price target is $79.00, and the average price target is $68.60. MIRM is currently trading a few cents above the low-end target. 

Technical tailwind: MIRM is showing technical strength and high levels of institutional demand.

It’s trading above the 20-, 30-, 50- and 100-day moving averages, with an RSI of 63.53.

This indicates that bullish momentum could be building ahead of next month’s Q2 earnings report.

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Bear Case 

Key risk: The primary risk for Mirum Pharmaceuticals (MIRM) stock stems from its heavy reliance on a narrow product portfolio, particularly its flagship drug, LIVMARLI. 

It currently accounts for a significant majority of current revenues.

If uptake slows, competitors enter the space, or reimbursement issues arise, earnings could be sharply impacted.

Likewise, any negative safety, efficacy, or commercial news related to LIVMARLI would disproportionately hurt Mirum’s valuation.

Macro/sector headwinds: Biotech stocks are highly sensitive to clinical trial results. A failed or delayed trial would likely result in sharp losses.

Several pipeline programs are in mid-to-late-stage development. Mirum’s future growth is tied to positive trial results and FDA approvals from those programs.

Competitive threat: Mirum’s biggest direct competitor is Ipsen.

Its BYLVAY medication is approved for the treatment of liver itching in patients with Progressive Familial Intrahepatic Cholestasis (PFIC).

It has also gained approval for Alagille syndrome (ALGS) in the U.S. and EU, directly threatening LIVMARLI’s market share.

Ipsen has a more extensive global commercial infrastructure, enabling faster international rollout and deeper payer access.

Head-to-head comparisons could influence physician preference depending on safety, efficacy, and formulation differences.

Crowded-trade concern: International expansion and transitioning to new formulations (like tablet forms) present operational and regulatory hurdles.

Underperformance in any major market could undermine bullish revenue targets and result in substantial losses.

Quick Checklist 

✅ Thesis still valid after today’s close
✅ Volume confirms move above key levels
✅ Catalyst date double-checked (July 16, 2025)

That’s all for today’s Everyday Alpha. We’ll have a new pick for you every morning before the market opens, so stay tuned!

Best Regards,
—Noah Zelvis
Everyday Alpha