Velorum Therapeutics

What do they actually do

Velorum Therapeutics is a preclinical biotech that engineers protein drugs to bind and remove heme or heme intermediates (porphyrins) from the blood. The company does not have a marketed product; current work is in discovery, in vitro studies, animal models, and IND‑enabling activities ahead of first‑in‑human trials (company pipeline, YC company page).

Its lead oncology program (VEL‑03) is a heme‑sequestering biologic being advanced for non‑small cell lung cancer and other solid tumors, with plans for first‑in‑human testing after GLP tox and GMP manufacturing are completed. In parallel, Velorum is developing a porphyrin‑scavenging biologic aimed at directly removing protoporphyrin IX (PPIX) for patients with EPP/XLP, a rare phototoxic disorder (NIH RePORTER grant summary for VEL‑03, company pipeline, launch post).

Who are their target customer(s)

• Oncology clinics and medical oncologists treating solid tumors: Need new options that slow tumor growth or sensitize tumors to standard treatments in resistant/aggressive disease; few reliable mechanisms beyond existing chemo, radiation, and immunotherapy (pipeline, NIH grant).
• Patients with EPP/XLP and their treating physicians: Severe, life‑disrupting light sensitivity from porphyrin buildup with limited available treatments; desire for therapies that reduce circulating PPIX and improve daily function (pipeline).
• Biotech/pharma BD teams seeking oncology combination assets: Look for differentiated early‑stage mechanisms to pair with standard of care but worry about robustness of preclinical data, safety margins, manufacturability, and biomarker strategy to de‑risk deals (NIH grant, company news).
• Clinical trial sponsors and investigator groups for first‑in‑human studies: Require IND‑ready candidates with GLP tox, reproducible GMP supply, and clear biomarker/PK‑PD plans to run small safety and dose‑finding trials efficiently (pipeline, YC page).
• Rare‑disease centers and patient advocacy organizations: Need therapies that meaningfully lower porphyrin levels and practical support for recruiting and executing small, specialized trials in sparse patient populations (pipeline, about page).

How would they acquire their first 10, 50, and 100 customers

• First 10: Target ~7 academic oncology centers and ~3 EPP/XLP specialty clinics with concise preclinical and tox data, proposing funded pilot or investigator‑initiated Phase 1 collaborations with IND‑grade supply and protocol support (pipeline, NIH RePORTER).
• First 50: Expand outreach to ~40 regional oncology centers and ~10 CROs via specialty‑meeting presentations and preclinical publications, offering centralized trial enablement (templates, biomarker assays, single‑contracting playbook) to speed onboarding.
• First 100: Add community oncology networks, specialty pharmacies, and rare‑disease groups through webinars, investigator roadshows, and registry integrations to drive referrals; in parallel, run targeted BD for combo trials leveraging manufacturing slots, CRO ties, and a growing safety database.

What is the rough total addressable market

Top-down context:

Two markets: a small, concentrated rare‑disease segment (EPP/XLP) and a much larger but uncertain oncology segment (advanced solid tumors, starting with NSCLC). These are Velorum’s stated targets (pipeline, NIH RePORTER).

Bottom-up calculation:

EPP/XLP prevalence is commonly cited at ~1:75,000 to 1:200,000; applied to ~335M U.S. population implies ~1,700–4,500 affected individuals, with diagnosed cases likely at the low end (peer‑reviewed review, NORD). For oncology, the U.S. sees roughly 229k new lung cancers annually and an estimated ~160k people living with metastatic lung cancer; initial addressable patients are a late‑line NSCLC subset, not the full incidence (ACS, metastatic cancer prevalence study).

Assumptions:

• U.S. population ~335M for rare‑disease prevalence math.
• Initial oncology use begins in late‑line metastatic NSCLC and/or biomarker‑defined subsets, not all lung cancer patients.
• Combination use and biomarker requirements will narrow early addressable oncology populations despite larger overall incidence.

Who are some of their notable competitors

• CLINUVEL (SCENESSE/afamelanotide): Approved implant to reduce phototoxicity in adult EPP; sets standard of care and payer pathways in EPP while acting via photoprotection rather than porphyrin removal (CLINUVEL PDF).
• Disc Medicine (bitopertin): Oral GlyT1 inhibitor that lowers PPIX; completed two Phase 2 EPP/XLP studies and filed an NDA with a Phase 3 ongoing, representing a potential disease‑modifying competitor in EPP/XLP (program page, APOLLO trial).
• Tanabe Pharma (dersimelagon/MT‑7117): Oral MC1R agonist that increases melanin; showed increased symptom‑free sunlight exposure in EPP/XLP and has reported positive topline Phase 3 results, positioning it as another late‑stage EPP option (NEJM study, press release).
• CSL Behring (CSL889 hemopexin): Plasma‑derived hemopexin to scavenge free heme (in early clinical development for sickle cell VOC); validates the heme‑sequestration therapeutic concept relevant to Velorum’s oncology mechanism (review on scavenger protein therapeutics, CSL889 trial).