The Cancer Target Nobody Could Hit for 40 Years Just Got Hit Again

The data come from the Phase 1/2 TARGET-D 101 trial, with a cutoff of October 7, 2026. The headline numbers in pancreatic cancer are genuinely encouraging:

• In second-line PDAC (patients who had already failed one prior therapy) at the 900 mg daily dose, 50% of patients had confirmed tumor shrinkage. That's 4 out of 8 patients.
• Broadening to second-line and beyond, the confirmed overall response rate was 41% (9 out of 22 patients), including two complete responses (one confirmed and one unconfirmed, with treatment ongoing).
• Even in heavily pretreated patients (third or fourth line), 36% still responded.

The drug also showed activity beyond the pancreas. In non-small cell lung cancer at a lower 600 mg dose, the confirmed response rate hit 43% (10 out of 23 patients). Early colorectal cancer data are trickling in too, though the patient numbers are still small.

Safety looked manageable. Verastem reported mostly low-grade gastrointestinal side effects and low rates of patients dropping out due to toxicity.

Small Numbers, Big Caveat

Before you get too excited, a reality check: these are early-phase results from small patient groups. The key efficacy readouts came from 22 evaluable pancreatic cancer patients and 23 lung cancer patients, not 200. The 200-plus figure refers to the broader enrollment across all tumor types and doses in the ongoing study.

Small datasets can swing wildly. One or two extra responders (or non-responders) would meaningfully change those percentages. That said, the consistency across lines of therapy and across tumor types is what caught analysts' attention.

Wall Street's Verdict: Cautiously Fired Up

The market reaction was decisively positive. Multiple analyst firms reiterated or raised their ratings on Verastem after the data dropped on October 14.

B. Riley bumped its price target to $19 from $16. RBC Capital also moved to $19 from $14. H.C. Wainwright kept a Buy rating with an $18 target, and BTIG described VS-7375 as a leading KRAS G12D program.

The bullish camp sees potential for a best-in-class drug. The more cautious voices (Mizuho held targets around $13 to $14) pointed to tolerability questions compared to the leading competitor, Revolution Medicines' zoldonrasib. Verastem also capitalized on the momentum with a roughly $250 million follow-on equity raise, building a war chest for the next phase of development.

The Race Is Getting Crowded

Verstem isn't alone in chasing KRAS G12D. Revolution Medicines has zoldonrasib, which reported a 30% response rate and 80% disease control rate in 40 evaluable pancreatic cancer patients. Astellas, Incyte, Hengrui, and Lilly all have programs in various stages too.

Verastem's 41% response rate in second-line-plus PDAC compares favorably to Revolution's 30%, though cross-trial comparisons are notoriously unreliable (different patients, different timepoints, different definitions of success). Still, it's the kind of number that earns you a seat at the table.

The company plans to launch a Phase 3 trial in first-line metastatic pancreatic cancer by the end of 2026. That's the real test. Phase 3 is where drugs either prove themselves against the standard of care or quietly disappear.

Why This Matters Beyond the Stock Price

Pancreatic cancer kills more than 50,000 Americans a year, and the five-year survival rate remains among the lowest of any major cancer. For decades, the field has been defined by incremental improvements in chemotherapy regimens; not the kind of targeted therapy revolutions seen in lung cancer or melanoma.

KRAS G12D inhibitors represent the first real chance to change that equation. The fact that multiple companies are racing toward Phase 3 trials means patients may finally have options tailored to their tumor's specific molecular defect, not just another round of chemo.

We're still in the early innings. The patient numbers are small, the follow-up is short, and no one has crossed the Phase 3 finish line yet. But the trajectory is unmistakable: the protein that scientists called undruggable for 40 years now has an entire competitive landscape of companies trying to drug it. For pancreatic cancer patients, that's the most hopeful sentence written in a very long time.

BMS Just Paid $1.5 Billion for a Company With Zero Approved Drugs

Bristol Myers Squibb is dropping $1.5 billion in cash on Orbital Therapeutics, a preclinical-stage startup with zero approved drugs and zero revenue. The bet says everything about Big Pharma's platform fever and the desperate race to fill patent cliffs.