MedPacto

Latest Trends in Drug Development

Trends in Anticancer Drug Development

Strong need for new treatments that can overcome the limitations of existing anticancer drugs
High medical unmet needs continue to rise despite technological advances (low response rates, high relapse rates, development of drug resistance)

Existing anticancer drugs

Next-generation anticancer drugs

Solely Tumor-focused

Immunotherapies, chemotherapies and targeted therapies target only the tumor cells

Importance of Tumor Microenvironment (Tumor Microenvironment)

Tumor stroma creates an environment that enables the growth and progression of malignant cells
Thus, cancer growth, metastasis and anticancer drug resistance can be circumvented by controlling the tumor microenvironment

One-fits-all Medicine

Low responses rates and severe adverse events due to treatment without patient screening

Biomarker-driven
Precision Medicine

Patients whose tumor expresses a specific biomarker receive the drug, regardless of the type of cancer

Single Blockbuster Drug

Conventional strategy of pursuing blockbuster medicines

New Age of
Combination Therapy

Field of cancer treatment is focused on combining a tumor-targeting anticancer agent and a drug that modulates the tumor microenvironment

Importance of the tumor microenvironment: Cancer cells secrete substances that regulate the tumor’s surroundings (tumor microenvironment) to promote cancer growth, metastasis and cancer stem cell formation, and suppress immune activity.(TGF-β1 is one of the most common substances)

Paradigm Shift in Clinical Trial Design

Biomarker-based therapy is the key to personalized medicine

Conventional Trial Design : Low response rates in each indication

Patients of
specific cancer indication
Drug to treat
specific cancer indication
Low treatment
responses observed

New Trial Design Paradigm : Biomarker-based Precision Medicine

Gene expression level, gene mutation, and protein analysis regardless of cancer type
Biomarker based
personalized treatment
High response rates
by personalized therapy

Adaptive trial design maximizes the value of anticancer drugs

Traditional Clinical Trials : Fixed Model

Biomarker-positive Biomarker-negative patients enrolled → Higher trial costs, longer development time / Lower probability of trial success

Adaptive (‘Seamless’) Clinical Trials : Flexible Model

Only biomarker-positive patients enrolled → Statistical validation with small number of patients, Preliminary clinical data can be used for NDA/BLA approvals

Status of Biomarker-based Anticancer Drug Development

Accelerated development of innovative drug to treat patients with
specific biomarkers, regardless of tumor tissue type

Value of Innovative Drugs

Anticancer drugs targeting mutations in specific genes are likely to drive market changes

“The fast market approval process encourages the development of innovative new drugs that target and treat only patients with certain genetic variations (biomarkers), regardless of tissue type”

Biomarker-based Drug Approvals (three FDA approvals to date)

Keytruda
(pembrolizumab)

Trade
Name (INN)Keytruda (pembrolizumab)

BiomarkerMSI-H or dMMR/TMB-H(Tumor Mutation Burden≥10mut/Mb)

Size of
Clinical Trials149 (5trials)

FDA Approval2017. 5 Approved for 18 cancer types, 32 indications(as of 2021)

Notes2022 Anticancer Drug Revenue Forecast No.1 ($19.7B)($11.1B in 2019, $14.4B in 2020)
Vitrakvi
(larotrectinib)

Trade
Name (INN)Vitrakvi (larotrectinib)

BiomarkerEVT6-NTRK Gene fusion

Size of
Clinical TrialsEffocacy: 55 Stability: 176
(3 trials)

FDA Approval2018. 11

Notes$8B USD ($7.5B) acquisition by Eli Lilly
Rozlytrek
(entrectinib)

Trade
Name (INN)Rozlytrek (entrectinib)

BiomarkerNTRK gene fusion

Size of
Clinical Trials54 (3 trails)

FDA Approval2019. 8

NotesIncresing number of global pharma companies are licensing candidates and pursuing acquisitions

MEdPACTO

Development of treatment
for patients

Development of treatmentfor patients

Development of treatment
for patients