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Myotonic Dystrophy Type 1: 3 biotech companies with clinical-stage programmes

Neurology & PsychiatryOrphan cornerData as of 2026-09-04

Competitors3in the clinic
Median market cap$250Muniverse median $1.20B
Allocated capital$2.0B$12.7B competing
Phase 3 or filed11 mid, 1 early
Pure plays1this indication only
Capital rank95/299crowding rank 65

Myotonic Dystrophy Type 1 sits in the orphan corner of the map: 3 developers, none of them large, with a median market cap of $250M against a $1.20B universe median.

A thin field of small caps usually means a narrow addressable population, so the whole field can be repriced by one readout or one financing.

ARWR alone accounts for 96% of the $12.7B of market capitalisation competing here, so this is a leader plus a tail rather than a level race.

$2.0B of allocated capital sits in Myotonic Dystrophy Type 1, 95th of 299 indications, and 1 of the 3 companies are pure plays that trade on this indication alone.

Companies developing drugs for Myotonic Dystrophy Type 1 3

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
RNAAtrium TherapeuticsAOC 1001Phase 32$250MNo3 31 Dec 2026
PEPGPepGenPGN-EDODM1Phase 22$220MYes1 30 Nov 2026
ARWRArrowhead PharmaceuticalsSRP-1003 InfusionPhase 12$12.2BNo7 31 Dec 2026

Sorted by phase, then by market capitalisation. Ticker links open the company profile. ARWR holds 96% of the $12.7B competing in this indication.

Why Myotonic Dystrophy Type 1 is a orphan corner

A thin field of small caps. Few competitors, none of them large.

The map splits indications on two folds: six or more competitors counts as crowded, and a median company above $1.20B counts as richly priced. Myotonic Dystrophy Type 1 has 3 competitors and a median of $250M.

Neurology & Psychiatry

Neurology & Psychiatry holds $120B of the map's capital, 17.9% of the total, spread across 46 indications and 58 companies. 19 of those indications have more than one company competing.

See every Neurology & Psychiatry indication on the map

Related indications in Neurology & Psychiatry

Multifocal Motor Neuropathy1 company · $2.0B · median $5.9BUncontestedAutism Spectrum Disorder2 companies · $1.9B · median $2.8BConcentrated betHuntington's Disease2 companies · $2.4B · median $2.8BConcentrated betGeneralized Anxiety Disorder1 company · $1.7B · median $5.1BUncontestedDementia With Lewy Bodies1 company · $1.6B · median $4.8BUncontestedParkinson's Disease5 companies · $2.5B · median $1.3BConcentrated betNarcolepsy Type 11 company · $2.6B · median $7.9BUncontestedTonic-Clonic Seizures1 company · $1.5B · median $5.8BUncontested

Myotonic Dystrophy Type 1 pipeline questions

Which biotech companies are developing drugs for Myotonic Dystrophy Type 1?

Atrium Therapeutics (RNA, AOC 1001, Phase 3); PepGen (PEPG, PGN-EDODM1, Phase 2); Arrowhead Pharmaceuticals (ARWR, SRP-1003 Infusion, Phase 1). Those are the 3 US-listed developers in the $100M to $20B market cap band with a clinical-stage programme in Myotonic Dystrophy Type 1.

How advanced is the Myotonic Dystrophy Type 1 pipeline?

Of the 3 clinical-stage programmes in Myotonic Dystrophy Type 1, 1 is in Phase 3 or filed, 1 is in mid-stage development and 1 is in early-stage development.

Which company leads the Myotonic Dystrophy Type 1 field?

ARWR is the largest developer in Myotonic Dystrophy Type 1, holding 96% of the $12.7B of market capitalisation competing in the indication. The median competitor is worth $250M.

When is the next Myotonic Dystrophy Type 1 catalyst?

The next dated catalyst in Myotonic Dystrophy Type 1 is PEPG on 30 Nov 2026. 3 dated events across the field fall in the next 12 months.

How this page is built

  • Universe is US-listed drug developers with a $100M to $20B market capitalisation, identified by SEC SIC code 2833, 2834 or 2836 or by a pharmaceutical or biotechnology industry classification, less a reviewed exclusion list of contract research organisations, device and diagnostics makers and diversified generics manufacturers.
  • An asset attributed to three or more different companies is treated as shared standard of care rather than any one company’s programme and excluded. Pembrolizumab alone appears under 21 tickers in raw trial data.
  • A programme counts only where at least one industry-sponsored trial supports it, so investigator-initiated academic studies of third-party drugs are excluded.

Allocated capital divides each company’s market capitalisation evenly across the indications it is running clinical programmes in, so every dollar of the universe is counted exactly once. Total market capitalisation competing counts a company in each of its indications and therefore sums to more than the universe.

Third-party market size estimates are not used anywhere on this page. Approved incumbents are excluded from competitor counts, and companies above $20B are outside the universe by construction.

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