Thread by @Pharmdca

$XFOR #CONVICTION Long Term position part 1
Pipeline:- Company lead drug Mavorixafor, an oral drug , works as an antagonist of the CXCR4 receptor for severe chronic neutropenia disorders including WHIM syndrome.
Current market cap ~ 240 million

$XFOR part 2
A quick recap of chronic neutropenia Neutrophils, type of white blood cells, an army that plays an important role in fighting off infections. Basically, lower the # of neutrophil count, the lower the ability of the body to fight infections (⬆️risk of infections)

$XFOR part 3
What number constitutes various forms of neutropenia?
ANC (Absolute Neutrophil Count) measures the number of neutrophils in the blood and this number basically is used to make an estimation of body's ability to fight off infection

$XFOR part 4
Quick recap of ANC count from a normal healthy person to severe neutropenia disorder
Normal neutrophil count= ANC No. between 1500 or higher
Neutropenia= ANC No. < 1000
Moderate= ANC between 500-1000
Severe= ANC < 500 (WHIM INDICATION)

$XFOR part 5
What are common causes of Neutropenia?
1) Congenital (mutations): Mutations in CXCR4 gene causing combined immunodeficiency and a congenital neutropenic disorder called WHIM SYNDROME
2) Chemotherapy
3) Viral infections like CMV
4) Drug induced neutropenia.

$XFOR part 6
What if these patients not treated?
-WHIM Syndrome: Majority of patients develop HPV-associated cancers as they age
-Neutropenia, if severe, increases the risk of life-threatening infection.
-Recurring infections even ones with normal gut flora and in the mouth

$XFOR part 7
Current Treatment regimen for CN and or WHIM
- G-CSF (Granulocyte-colony stimulating factor)- Standard of Care
- Hematopoietic stem cell transplantation
- Antibiotics
- Corticosteroids
- Immunosuppressive drugs
- White cell transfusions

$XFOR part 8 Brief review of G-CSF
- Current SOC for CN and WHIM
The standard treatment for chronic neutropenia is G-CSF
-manufactured hormone that causes the bone marrow to make and release neutrophils, thus improving a person's ability to fight off infections
-SubQ inj.

$XFOR part 9
Why the need for newer therapies for CN
- G-CSF requires daily subq injections
- Inconvenient
- Takes long time before optimal dosing
- Frequent treatment related side effect with G-CSF like bone pain (common side effect
- can cause secondary malignancies (AML)

$XFOR part 10
Advantages of Mavorixafor over G-CSF (current SOC)
- Mavorixafor is the first oral drug being developed for both WHIM and CN
-ORAL vs SubQ
- Better patient compliance
- Well tolerated drug.
- Very strong PH3 results

$XFOR part 11
Let's discuss Positive PH3 data for WHIM Syndrome - Hit both primary and secondary endpoints
- ANC of mavorixafor-treated patients remained above a clinically meaningful threshold of 500 cells/mL for 15.04 hours compared to 2.75 hours in the placebo group

$XFOR part 12 PH3 results
- ALC of mavorixafor-treated patients remained above a clinically meaningful threshold of 1,000 cells/mL for 15.80 hours compared to 4.55 hours in the placebo group
- ~60% reduction in annualized infection rate seen in the mavorixafor arm vs. placebo.

$XFOR part 13 PH3 results
- >75% reduction in the percentage of individuals experiencing severe infections (Grade 3 or higher) in the mavorixafor group vs. placebo group
- >70% reduction in mean total days with infections (2 weeks on mavorixafor treatment vs. 7 weeks on placebo

$XFOR part 14 PH3 results in nutshell for mavorixafor vs placebo
- Huge reduction in infection rate (60%)
- Big reduction in severity of infections (75%)
- Big reduction in duration of infections (70%)
- very well tolerated drug with no drug-related serious adverse event

$XFOR part 15
What's next after strong positive results for WHIM SYNDROME
- Company on track to submit NDA for mavorixafor early in 2H 2023 following completion of pre-NDA meeting with FDA
- Upon FDA approval, eligible for ~ 100 million prv voucher
- CN upcoming data
- Deal?

$XFOR part 16
Positive results for WHIM SYNDROME and its implications for upcoming data for Chronic Neutropenia late Q2 or early Q3
- Strongly believe positive results for WHIM likely translates to +ve results for CN ➡️Big indication
- WHIM RESULTS➡️+ve data for CN

$XFOR part 17 Neutropenia market
- market expected to grow over time due to rise in incidence of cancer cases worldwide
- novel therapy likely boost the market growth
- The global g-csf market grew from $7.99 billion in 2022 to $8.43 billion in 2023 and expected to rise

$XFOR Pipeline

$XFOR part 18 Conclusion
Disclaimer: This is my top 3rd long term position
Target: My price target changed after better than expected data last week for WHIM SYNDROME
I believe long term stock could hit between $5-$10+ and that's ~150-400% returns from here
Do your DD always