Biotech Pharma Investor

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Launched 6 months ago

Biotech Pharma Investor

Biotech Pharma Investor is an analysis-focused Substack for biotech investors.

Recent Notes

ENGN preliminary notes

Data update is terrible. Low durability is a big problem vs competition. But risk reward is favorable at current valuation:

• Market cap at $1.8 is $119M. Assuming cash burn will continue at $31M per quarter, ENGN will end 2026 with close to $200M. Note cash runway into 2H 2028.
• The underappreciated catalyst: a readout from the next cohort using a surfactant bladder rinse which is expected to increase efficacy.
• Data for newly assessed patients (since Oct 2025) considerably worse.

LRMR notes

Currently a single asset- single indication company (although I believe the platform could be used in other indications eventually).

• The company is developing a daily SC treatment that replaces the missing frataxin protein in mitochondria.

What I like

• Big catalysts soon
• OLE topline in Q2

QNRX update

• FDA feedback suggests single phase 3 enough for approval.
• FDA open on alternative design, but seems like not baseline-controlled designs (which to my understanding means the deadline for Series H warrants is not triggered).

$QNRX

Summary:

• By far the most advanced in clinical development Netherton treatment. Well-funded after private placement announcement in October.

$KPTI

Phase 3 in MF failed to meet the TSS co-primary. So this is in no way a clear win:

• Faster and clear SVR35 reduction.
• Evidence of disease modification.
• Clear reduction in OS (large effect size + p 0.022).

$DTIL

Interesting call on DMD:

• Lowest vector dose (matching SLDB, half of that by RGNX, 30% lower vs SRPT).

• 90% full capsid ratio.

First note on $BHVN

I am most interested in BHVN due to their peripheral degraders platform that can remove pathogenic proteins. But the potential of the rest of the pipeline is very interesting. Cash situation not great. But many data this year. And IgAN is a very large TAM + the program seems very de-risked based on preliminary data.

Financials

• Ended 2025 with $322.0 million. Subsequent raised another $136M (accounting for the $42.7 million payment to Knopp).

$FATE

Overview: Note that FATE started as an NK company but is now focused on CAR-Ts. Their tech combines the best attributes of various approaches:

1. Allogeneic
2. iPSC-derived (vs from donors)

KYBN

I took a quick look. May be good for a trade (Market cap<<cash, runway into 2028, prostate cancer interim readout in 2H) but I am not particularly excited by the pipeline and company’s history.

• Pipeline is early.
• Preliminary results in prostate cancer are not particularly exciting vs fierce competition + safety not great.
• Roxadustat for anemia in low-risk MDS is interesting efficacy-wise (+oral).

$CRMD

Market seems to be ignoring the Rezzayo potential. I think an once-weekly drug that simultaneously covers candida, aspergillus, and pneumocystis is very useful. It avoids AEs/interactions associated with azole + trimethoprim/sulfamethoxazole prophylaxis. Readout late Q2 2026. Goal is non-inferiority in 90d fungal-free survival (I think high probability of success).

$ANTX

• EBO for PV.
• Very interesting addition to the pipeline. Exploiting the observation of predictable, reversible dose-dependent reduction in Hematocrit.