Stocktwits Pharma Pulse: Roche Faces FDA Decision, Gene-Therapy Stocks Step Into Focus — The Week’s Biotech Watchlist

Rocket, RegenXBio, Precision BioSciences, Kyverna and uniQure will present on Monday at a cell and gene-therapy conference.

• Lilly won FDA approval last week to expand Jaypirca’s use to patients starting treatment for certain blood cancers.
• Ocular will present eye-treatment research between Oct. 8-9 ahead of an upcoming fourth-quarter FDA filing.
• Roche awaits an Oct.9 FDA decision on Tecentriq for certain colon-cancer patients after surgery.

After Eli Lilly (LLY) secured a new approval for its leukemia drug Jaypirca on Friday, the pharma spotlight turns this week to Roche’s bid to bring immunotherapy into postoperative colon-cancer treatment. Alongside the FDA milestone, Ocular Therapeutix (OCUL) and several gene-therapy developers will present research and program updates at conferences.

The Health Care Select Sector SPDR Fund (XLV) fell 2.7% last week, underperforming the broader market’s SPDR S&P 500 ETF Trust (SPY), which slipped 0.2%, and the Invesco QQQ Trust (QQQ), which gained 0.7%. The iShares Biotechnology ETF (IBB) declined 2.1%, while the SPDR S&P Biotech ETF (XBI) fell 0.4%. Both biotech funds outperformed XLV but lagged SPY and QQQ. 

Date	Ticker/Event	Development
Oct.5-7	Meeting on the Mesa	Cell and gene-therapy companies gather in Phoenix for presentations and regulatory discussions.
Oct.5	RCKT, RGNX, DTIL, KYTX, QURE	Rocket, RegenXBio, Precision BioSciences, Kyverna and uniQure have company spotlight presentations.
Oct.6	Nanoscope	Presentation on its retinal gene-therapy program under FDA review.
Oct.7-10	NACFC	The North American Cystic Fibrosis Conference takes place in Atlanta.
Oct.8-9	OCUL	Axpaxli and SOL-1 presentations at ophthalmology events.
Oct.9	Roche, FDA	Target action date for Tecentriq in a defined postoperative colon-cancer population.
Oct.9,11	ENTX, ASBMR	Entera presents Phase 2 analyses of its oral osteoporosis treatment EB613.
Oct.11-12	HFSA	Late-breaking heart-disease presentations extend into the following week.

LLY Starts October With Jaypirca Approval

Lilly’s latest FDA approval expands Jaypirca’s use to patients starting treatment for chronic lymphocytic leukemia or small lymphocytic lymphoma whose cancer lacks a genetic abnormality known as a 17p deletion.

The decision was supported by Bruin CLL-313, a randomized study of 282 patients in which Jaypirca reduced the risk of disease progression or death by 80% compared with Bendamustine plus Rituximab, a chemotherapy-based treatment.

Patients receiving the comparator went a median of 33.5 months without their disease worsening. The Jaypirca group had not reached that median by the analysis, meaning more than half remained alive without disease progression. 

RCKT, QURE, RGNX Lead Gene-Therapy Watchlist

Meeting on the Mesa, an annual industry conference focused on cell and gene therapies, opens on Monday at the Arizona Biltmore in Phoenix and runs through Wednesday. RegenXBio (RGNX), Rocket Pharmaceuticals (RCKT), Precision BioSciences (DTIL), Kyverna Therapeutics (KYTX) and uniQure (QURE) are scheduled to present on Monday. 

Monday’s agenda also features a keynote from Karim Mikhail, director of the FDA center that oversees cell and gene therapies. Bristol Myers Squibb and Kite executives will discuss access to CAR-T treatments, which modify immune cells to fight cancer, while regulators from the U.S., U.K. and Japan will also discuss how cell and gene therapies are evaluated and approved. 

OCUL Presents Axpaxli Data Ahead Of FDA Filing

Ocular Therapeutix (OCUL) will present research on its experimental eye treatment Axpaxli on Thursday and Friday at events surrounding the American Academy of Ophthalmology meeting.

The presentations include findings from SOL-1, a Phase 3 trial in wet age-related macular degeneration, a condition in which abnormal blood vessels damage the retina and can cause central vision loss. Ocular has already released the trial’s headline results, with this week’s sessions offering a closer look at the findings ahead of an upcoming FDA application.

The company plans to file in the fourth quarter using SOL-1’s efficacy and safety results, alongside interim safety data from its separate SOL-R trial. SOL-R is still underway, with headline results expected in the first quarter of 2028.

Roche Awaits Friday’s Colon-Cancer Decision

Roche faces an Oct.9 FDA target date for its bid to expand Tecentriq and Tecentriq Hybreza into treatment after surgery for certain patients with stage III colon cancer.

The application covers use with chemotherapy in patients whose tumors have defects in repairing DNA, known as mismatch-repair deficiency or high microsatellite instability. These features help identify cancers that may respond to immunotherapy.

The supporting Atomic study enrolled 712 patients. At three years, 86.3% of patients receiving the immunotherapy-containing treatment were alive without cancer recurrence, compared with 76.2% in the control group. The treatment reduced the risk of recurrence or death by 50%.

Entera’s EB613 Joins Weekend Research Lineup

Entera Bio (ENTX) will present analyses of its Phase 2 study of EB613, an experimental oral osteoporosis treatment, on Oct.9 and Oct.11 at the American Society for Bone and Mineral Research’s annual meeting. 

Meanwhile, the Heart Failure Society of America’s meeting brings more research updates on Oct.11 and Oct.12. Sunday’s presentations include patient health-status findings from the Cardio-TTRansform study of Eplontersen and Phase 3 Reveal results for an imaging test designed to detect abnormal protein deposits in the heart. Monday features ACACIA-HCM, a trial of Aficamten in patients with an abnormally thickened heart muscle that does not obstruct blood flow. 

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