Oral small molecule drugs in Phase 1 and Phase 2: North America compared with Europe

981 oral small molecule drugs in early clinical development, split between the United States and Canada and the European market, compared on development signals, planned trials, therapeutic area, sponsor size, and sponsor funding.

771
Drugs, US and Canada
210
Drugs, Europe
662
Sponsor companies
2
Years of signal history

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How to read this report

Source
All figures come from Zymewire. No external databases, registries, or estimates were used.
Coverage
Orally administered small molecule drugs whose most advanced phase is Phase 1 or Phase 2, with activity in the past two years.
Regions
Defined by sponsor headquarters. The two regions are separate datasets, so a drug appears in one only.
Signals
Counted over the trailing twelve months. Because the two regions differ in size, comparisons are shown as a share of each region.
Sponsor fields
Sponsor size, funding, and pipeline depth describe the company, not the individual drug.
Funding
Disclosed funding events summed over this year and last. No figure means no disclosed event in that window.

What this report covers

01 / Signal momentum

Where announced intent and disclosed results are concentrated by phase

Future plan signals show where sponsors are declaring what they intend to run next. Result disclosures show where data has just landed. Reading each against the drug's phase shows which end of early development is moving.

Oral small molecules  /  Phase 1 and Phase 2  /  past 12 months
Future plan signals

Share of drugs at each phase with a future plan on record in the last year

Drugs with a future plan on record in the last year, by phase and region
PhaseUS and Canada countUS and Canada shareEurope countEurope share
Phase 1 drugs12434.0%2739.7%
Phase 2 drugs11929.3%5035.2%
Result disclosures

Share of drugs at each phase that disclosed results in the last year

Drugs disclosing results in the last year, by phase and region
PhaseUS and Canada countUS and Canada shareEurope countEurope share
Phase 1 drugs15141.4%3145.6%
Phase 2 drugs18044.3%5740.1%
237
Phase 2 oral small molecules disclosed results in the past 12 months across both regions, 180 in the US and Canada and 57 in Europe
151
Phase 1 oral small molecules carry a future plan on record across both regions, 124 in the US and Canada and 27 in Europe

A future plan attached to a Phase 1 drug points to a Phase 2 being prepared. A result disclosure on a Phase 2 drug puts that drug at a Phase 3 decision. Together the two panels show how much of each region's early pipeline is approaching its next step, and at which end of it.

02 / Therapeutic area

Oncology is 40% of the North American pipeline and 27% of the European one

Each therapeutic area's share of its own region, shown as the gap between the two. Bars to the right run heavier in the US and Canada, bars to the left run heavier in Europe.

Oral small molecules  /  Phase 1 and Phase 2
Therapeutic area as a share of each region
Therapeutic areaUS and CanadaEuropeDifference in percentage points
Oncology40%27.3%+12.7
Mental Health8.6%6.2%+2.4
Metabolic & Endocrine5.5%3.3%+2.2
Hematology4%2.4%+1.6
Infectious Diseases4.3%3.3%+1.0
Liver Disease2.9%2.4%+0.5
Musculoskeletal0.8%1%-0.2
Dermatology2.1%2.4%-0.3
Pain3%3.8%-0.8
Cardiology2.2%3.3%-1.1
Gastroenterology3.3%5.3%-2.0
Respiratory2.5%4.8%-2.3
Autoimmune / Allergy3.9%6.7%-2.8
Reproductive Health0.1%3.3%-3.2
CNS14.2%19.1%-4.9

CNS runs five percentage points heavier in Europe, at 19.1% against 14.2%. Autoimmune and allergy, respiratory, and gastroenterology each run two to three points heavier in Europe. Areas with fewer than eight drugs across both regions are excluded.

Oral small molecules  /  Phase 1 and Phase 2  /  drug counts by region
US and Canada

Drugs by therapeutic area

Europe

Drugs by therapeutic area

Drugs by therapeutic area and region
Therapeutic areaUS and CanadaEurope
Oncology30757
CNS10940
Mental Health6613
Metabolic & Endocrine42Not in top areas
Infectious Diseases33Not in top areas
Hematology31Not in top areas
Autoimmune / Allergy3014
GastroenterologyNot in top areas11
RespiratoryNot in top areas10
PainNot in top areas8
03 / Conditions

The most common indications for oral small molecules in early development

The indications most often being treated by oral small molecules in early development, split into oncology and everything else. Oncology is separated because it would otherwise absorb most of a single ranking.

Oral small molecules  /  Phase 1 and Phase 2  /  both regions combined
Oncology indications

Most common specific cancer types

Most common specific cancer types, both regions combined
Cancer typeDrugs
Acute Myeloid Leukemia24
Breast Neoplasms13
Prostate Cancer12
NSCLC11
Ovarian Cancer8
Colorectal Cancer8
Myelodysplastic Syndromes5
Multiple Myeloma5
Pancreatic Cancer5
Hepatocellular Carcinoma4
Triple Negative Breast Neoplasms4
Melanoma4
All other indications

Most common indications outside oncology

Most common indications outside oncology, both regions combined
IndicationDrugs
Obesity31
Alzheimer Disease23
Parkinson Disease17
Pain16
Ulcerative Colitis10
Major Depressive Disorder10
Amyotrophic Lateral Sclerosis9
Schizophrenia8
Idiopathic Pulmonary Fibrosis8
Heart Failure6
Psoriasis6
Metabolic-associated Steatohepatitis6

Acute myeloid leukemia leads the oncology ranking at 24 drugs. Obesity leads outside oncology at 31, ahead of Alzheimer disease at 23 and Parkinson disease at 17. Eight of those obesity drugs act on a GLP-1 target, every one of them oral, in a category where the established products are injectable.

04 / Sponsor pipeline depth

Half of European drugs belong to sponsors who have never reached Phase 3

Every drug grouped by the most advanced phase associated with its sponsor's programs, across the sponsor's whole portfolio rather than this drug alone.

Oral small molecules  /  Phase 1 and Phase 2  /  grouped by sponsor portfolio

Most advanced phase associated with the sponsor's programs

Drugs by the most advanced phase associated with the sponsor's programs
RegionPhase 2 or earlierPhase 3Phase 4Total
US and Canada329326116771
Europe1096635210

51.9% of European drugs and 42.7% of North American drugs have a sponsor whose most advanced program sits at Phase 2 or earlier.

05 / Sponsor profile

Sponsor headcount and disclosed funding by region

The companies behind these drugs, by size and by capital raised over the past two years.

Oral small molecules  /  Phase 1 and Phase 2  /  sponsor companies
US and Canada

Sponsor size

495 companies by approximate employee count

Sponsor companies by approximate employee count and region
EmployeesUS and CanadaEurope
1 to 1016862
11 to 5023172
51 to 20019234
201 to 1,00011918
Over 1,0005523

Sponsor funding

Companies by funding disclosed over two years

Sponsor companies by funding disclosed over two years and region
FundingUS and CanadaEurope
No funding recorded261101
Under $10M5320
$10M to $50M5227
$50M to $200M7014
Over $200M595
06 / Rare disease

Around half of drugs in each region target a rare disease, but far fewer hold orphan status

Drugs targeting a rare disease compared with drugs holding an orphan designation. This measure cuts across therapeutic areas rather than sitting inside one: a drug for a rare cancer is recorded under oncology, and a drug for a rare neurological condition under CNS.

Oral small molecules  /  Phase 1 and Phase 2
Targets a rare disease Holds orphan status
Rare disease and orphan status by region
MeasureUS and Canada drugsEurope drugsUS and Canada shareEurope share
Targets a rare disease39811151.6%52.9%
Holds orphan status1305016.9%23.8%

The gap between the two measures is 34.7 points in the US and Canada and 29.1 points in Europe.

07 / Compound handling

37.9% of North American drugs are potent compounds, against 22.4% in Europe

Drugs identified as potent compounds, as a share of each region.

Oral small molecules  /  Phase 1 and Phase 2
Drugs identified as potent compounds, by region
RegionDrugsShare of region
US and Canada29237.9%
Europe4722.4%

That is 292 drugs in the US and Canada and 47 in Europe.

08 / Geography

Two states hold 59% of North American drugs. Europe's top two countries hold 36%

Sponsor location within each region, shown by state or province in North America and by country in Europe.

Oral small molecules  /  Phase 1 and Phase 2

US and Canada

Drugs by sponsor state or province

Drugs by sponsor state or province, US and Canada
State or provinceDrugs
California259
Massachusetts194
New York44
New Jersey41
Delaware31
Pennsylvania24
Texas16
Colorado16

Europe

Drugs by sponsor country

Drugs by sponsor country, Europe
CountryDrugs
United Kingdom42
Switzerland33
France19
Sweden18
Belgium16
Spain14
Germany14
Denmark10

Europe's activity is spread across 19 countries, with no single market holding more than a fifth of the regional total.

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