On May 18, 2024, Mirum Pharmaceuticals revealed important findings at the 56th ESPGHAN Annual Meeting in Milan, showcasing the efficacy of LIVMARLI® (maralixibat) in treating Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). Clinical and real-world data indicate significant improvements in patients’ liver health and reduction in pruritus and bilirubin levels. Moreover, real-world evidence shows a reduction in concomitant medication use for ALGS patients on LIVMARLI.

In Q1 2024, Mirum achieved $68.9 million in net product sales, a 137% increase from Q1 2023, and is on track to meet its annual revenue guidance. LIVMARLI received U.S. approval for PFIC, expanding its market presence. The company is also advancing volixibat for Primary Sclerosing Cholangitis (PSC) and Primary Biliary Cholangitis (PBC), with interim analyses expected in June 2024. Mirum reported a net loss of $25.3 million for Q1 2024, but increased its cash reserves to $302.8 million.

Breakthrough Data at ESPGHAN Annual Meeting

Long-term Benefits in PFIC

Abstract 587: Long-term Maintenance of Response and Improved Liver Health with Maralixibat in Patients with Progressive Familial Intrahepatic Cholestasis: 2-year Data From the MARCH-ON Study
Presented by Professor Richard J. Thompson, MD, King’s College London, United Kingdom.

Patients with PFIC treated with LIVMARLI for up to two years demonstrated significant and sustained improvements in pruritus severity, serum bile acid (sBA) levels, total bilirubin, and growth. These improvements were consistent even in patients initially randomized to placebo, who later received LIVMARLI in the open-label study.

Significant Reductions in Bilirubin Levels

Abstract 594: Maralixibat Leads to Significant Reductions in Bilirubin for Patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH Trial
Presented by Lorenzo D’Antiga, MD, Papa Giovanni XXIII Hospital, Bergamo, Italy.

Patients treated with LIVMARLI showed substantial decreases in both total and direct bilirubin levels compared to placebo. Notably, 40% of patients with abnormal bilirubin at baseline achieved normalization, versus none in the placebo group. These reductions in bilirubin levels aligned with consistent reductions in sBA levels.

Impact on Concomitant Medication Use in ALGS

Abstract 600: Maralixibat Impact on Concomitant Medication Use for the Treatment of Cholestatic Pruritus in Alagille Syndrome: Real-World Experience
Presented by Jolan Terner-Rosenthal, PhD, Mirum Pharmaceuticals, Inc., Foster City, California, USA.

Real-world data from 116 patients treated with LIVMARLI for at least one year indicated that over one-third of patients could discontinue more than one concomitant antipruritic medication, and one in five patients discontinued more than two medications. These reductions suggest that LIVMARLI may significantly reduce the polypharmacy burden within the first year of treatment.

Financial Performance and Strategic Progress

First Quarter 2024 Conference Call Highlights

On May 8, 2024, Mirum Pharmaceuticals hosted its first-quarter 2024 conference call, highlighting substantial progress across key strategic objectives to grow its commercial business, expand indications of commercial medicines, and advance volixibat to market.

Revenue and Sales Growth

Total net product sales for the first quarter of 2024 were $68.9 million, representing a 137% increase from the first quarter of 2023. LIVMARLI continues to perform robustly, with new diagnoses and prevalent patients coming to treatment in both the U.S. and internationally. Mirum is on track to meet its full-year revenue guidance of $310 million to $320 million, driven by increased demand, international launches, and contributions from PFIC approval.

LIVMARLI Approval and Expansion

In March 2024, LIVMARLI received U.S. approval for PFIC, marking a significant milestone for both Mirum and the PFIC patient community. This approval, backed by years of dedication from patients, researchers, and Mirum’s team, allows the company to bring LIVMARLI to patients with rare genetic types of PFIC. The pivotal data, recently published in The Lancet, highlighted improvements in itch, bile acids, bilirubin, and growth with LIVMARLI treatment.

Pipeline Advancements: Volixibat

Mirum is advancing volixibat in Primary Sclerosing Cholangitis (PSC) and Primary Biliary Cholangitis (PBC), leveraging scientific and regulatory insights from the LIVMARLI program. Interim analysis of volixibat in the VISTAS PSC and VANTAGE PBC studies is scheduled for June 2024, aiming to address bile acid accumulation in patients with these conditions. These studies represent significant opportunities, as there are currently no approved therapies for PSC and no approved therapies to treat cholestatic pruritus in PBC.

Financial Overview

Mirum’s total revenue for the first quarter of 2024 was $69.2 million, compared to $31.6 million in the first quarter of the previous year. Total operating expenses for the quarter ended March 31 were $95.7 million, including R&D expenses of $32.2 million, SG&A expenses of $45.6 million, and cost of sales of $17.8 million. Net loss for the quarter was $25.3 million, or $0.53 per share. Mirum’s cash, cash equivalents, and investments increased to $302.8 million as of March 31, 2024, up from $286.3 million at the end of 2023.

Insights

• LIVMARLI shows long-term effectiveness in treating PFIC.
• Significant reduction in bilirubin levels was observed in PFIC patients treated with LIVMARLI.
• Real-world data indicates LIVMARLI reduces the need for multiple medications in ALGS patients.
• Mirum Pharmaceuticals’ financial performance is robust, with significant revenue growth.

The Essence (80/20)

Core Topics:

1. Clinical Efficacy of LIVMARLI: LIVMARLI demonstrates sustained improvements in liver health and pruritus in PFIC and ALGS patients.
2. Financial Growth: Mirum Pharmaceuticals shows strong financial performance with significant revenue growth and robust market expansion.
3. Regulatory and Pipeline Advances: U.S. approval of LIVMARLI for PFIC and advancement of volixibat for PSC and PBC highlight strategic progress.

The Action Plan – What Mirum Pharmaceuticals Should Do:

1. Enhance Clinical Outreach: Increase engagement with healthcare providers to educate on the benefits of LIVMARLI for PFIC and ALGS.
2. Market Expansion: Focus on international launches and new patient diagnoses to drive revenue growth.
3. Pipeline Development: Accelerate volixibat clinical trials to bring new treatments for PSC and PBC to market.
4. Monitor Financial Health: Maintain fiscal discipline while investing in R&D to sustain financial growth.

Blind Spot

While the focus is on the effectiveness of LIVMARLI and financial success, monitoring long-term safety and patient adherence to treatment protocols is crucial to ensuring sustained benefits and minimizing potential side effects.

Mirum Pharmaceuticals (MIRM) Technical Analysis

Price and Moving Averages

• Current Price: $25.30
• 50-day Moving Average: $26.05
• 200-day Moving Average: $28.65

The price is currently below both the 50-day and 200-day moving averages, indicating a bearish trend.

Volume

• The volume is moderate with occasional spikes, which are not indicative of strong buying or selling pressure. The latest volume bar shows an increase, suggesting some renewed interest in the stock.

Relative Strength Index (RSI)

• Current RSIIn the world of technical analysis, the Relative Strength Index (RSI) stands as a cornerstone tool for traders seeking insights into market momentum. Developed by J. Welles Wilder ... More: 51.34

The RSI is around the mid-point (51.34), suggesting that the stock is neither overbought nor oversold. This level typically indicates a period of consolidation or indecision in the market.

On-Balance Volume (OBV)

• The OBVThe On Balance Volume indicator (OBV) is a technical analysis tool used to measure the flow of money into and out of a security over a specified period of time. It is a cumulative ... More line has been relatively flat, which means that the buying and selling volumes are fairly balanced. There’s no significant accumulation or distribution trend.

Stochastic RSI

• Current Value: 0.565

The Stochastic RSIIn the realm of technical analysis, the Stochastic RSI (StochRSI) emerges as a powerful tool for traders seeking to navigate market dynamics with precision. Developed by Tushar S. ... More is in the neutral zone. It doesn’t indicate an overbought or oversold condition, which aligns with the RSI’s indication of a consolidating market.

Average Directional Index (ADX)

• Current Value: 17.75

The ADXThe Average Directional Index (ADX) stands as a cornerstone indicator in the toolkit of technical traders, offering insights into the strength of market trends. Developed by Welles... More is below 20, suggesting that the current trend (whether up or down) is weak. This supports the idea that the stock is in a consolidation phase rather than a strong trend.

Chaikin Oscillator

• Current Value: -191,247

The negative value of the Chaikin OscillatorNamed after its creator Marc Chaikin, the Chaikin Oscillator stands as a formidable tool in the arsenal of technical analysts. This oscillator is designed to measure the accumulati... More suggests that there is more selling pressure than buying pressure. However, the magnitude is not extremely large, indicating moderate selling pressure.

Key Levels

• Support: $24.00 (recent lows)
• Resistance: $26.05 (50-day MA), $28.65 (200-day MA)

3-Month Outlook: Hold

• Given the current consolidation and lack of strong directional indicators, holding is recommended to observe if the stock breaks above the 50-day MA for a bullish signal or below the support level for a bearish signal.

6-Month Outlook: Hold

• Similar to the 3-month outlook, more data and trend confirmation are needed. Watching the volume and OBV for accumulation signs could provide early buy signals.

12-Month Outlook: Buy

• Long-term, if the stock can break above the 200-day MA and maintain higher levels, it may signal a bullish reversal. Given the current levels, a buy recommendation is cautious but optimistic for a turnaround over the year.

Overall, the stock is in a neutral phase with a potential for future bullish activity if it can overcome the immediate resistances.

Looking Ahead

Mirum Pharmaceuticals continues to make significant strides in advancing treatments for rare liver diseases, evidenced by the promising data presented at the ESPGHAN Annual Meeting and the company’s robust financial performance in the first quarter of 2024. With ongoing efforts to expand the indications of its commercial medicines and advance its pipeline, Mirum is well-positioned to continue delivering life-changing therapies to patients worldwide.

Mirum Pharmaceuticals FAQ

Book Recommendations

1. “The Innovator’s Prescription: A Disruptive Solution for Health Care” by Clayton M. Christensen, Jerome H. Grossman, and Jason Hwang – For insights on innovative healthcare solutions.
2. “The Emperor of All Maladies: A Biography of Cancer” by Siddhartha Mukherjee – To understand the impact and development of treatments for complex diseases.
3. “Bad Blood: Secrets and Lies in a Silicon Valley Startup” by John Carreyrou – For a perspective on the challenges and ethics in pharmaceutical development.