The Daily Biotech Pulse: Positive Adcom Verdict For Merck, Homology Medicine Slips On Mixed Gene Therapy Efficacy Data

Here's a roundup of top developments in the biotech space over the last 24 hours.

Scaling The Peaks

(Biotech stocks hitting 52-week highs on Dec. 17)

• Acceleron Pharma Inc XLRN
• Allergan plc AGN
• Amgen, Inc. AMGN
• Aptose Biosciences Inc APTO
• Aurinia Pharmaceuticals Inc AUPH
• Bio-Rad Laboratories, Inc. Class A Common Stock BIO
• Cue Biopharma Inc CUE
• Deciphera Pharmaceuticals Inc DCPH
• Globus Medical Inc GMED
• Horizon Therapeutics PLC HZNP
• Iovance Biotherapeutics Inc IOVA
• Ironwood Pharmaceuticals, Inc. IRWD
• Medtronic PLC MDT(announced acquisition of Klue, a software company focused on behavior tracking)
• Mirati Therapeutics Inc MRTX
• Momenta Pharmaceuticals, Inc. MNTA
• Nantkwest Inc NK
• Novo Nordisk A/S NVO
• Neoleukin Therapeutics Inc NLTX
• NuVasive, Inc. NUVA
• Ovid Therapeutics Inc OVID
• Prevail Therapeutics Inc PRVL( announced Orphan Drug Designation for its dementia asset)
• Principia Biopharma Inc PRNB
• Repro-Med Systems, Inc. KRMD
• ResMed Inc. RMD
• Soligenix, Inc. Common Stock SNGX
• SpringWorks Therapeutics Inc SWTX
• Turning Point Therapeutics Inc TPTX
• Veru Inc VERU

Down In The Dumps

(Biotech stocks hitting 52-week lows on Dec. 17)

• Achieve Life Sciences Inc ACHV
• AEterna Zentaris Inc. AEZS
• Aethlon Medical, Inc. AEMD
• Athersys, Inc. ATHX
• Brickell Biotech Inc BBI
• China SXT Pharmaceuticals Inc SXTC
• Endologix, Inc. ELGX
• Gossamer Bio Inc GOSS
• Miragen Therapeutics Inc MGEN
• MEREO BIOPHARMA/ADR MREO
• Phathom Pharmaceuticals Inc PHAT
• Therapix Biosciences Ltd – ADR TRPX
• Theratechnologies Inc (NASDAQ: THTX
• Tocagen Inc TOCA
• Wave Life Sciences Ltd WVE

Stocks In Focus

Homology Medicines Reports Mixed Efficacy Data For Gene Therapy To Treat Phenylketonuria

Homology Medicines Inc FIXX announced initial Phase 1/2 data from the pheNIX trial that evaluated its HMI-102 gene therapy in patients with phenylketonuria, with safety data from the Cohort 1 consisting of two patients administered low dose and Cohort 2 consisting one patient administered mid dose, showing that HMI-102 was well tolerated.

The efficacy data was mixed, with the Cohort 1 patients not showing a reduction in phenylalanine through Week 10 and 12. But the patient dosed in Cohort 2 showed a reduction in phenylalanine of 35% and 48% from baseline at Weeks 1 and Week 4, respectively.

The stock fell 21.84% to $17 in after-hours trading.

FTC Files Compliant To Block Illumina's Deal To Buy PacBio

The FTC said it has authorized an action to block Illumina, Inc.'s ILMN $1.2 billion buy of Pacific Biosciences of California PACB. In an administrative complaint, the agency alleged that "Illumina is seeking to unlawfully maintain its monopoly in the U.S. market for next-generation DNA sequencing systems by extinguishing PacBio as a nascent competitive threat."

The administrative trial is set to begin Aug. 18, 2020.

Pacific Biosciences shares declined 7.85% to $4.93 in after-hours trading.

See Also: After Amarin Snags Vascepa Label Expansion, Analyst Says Biopharma An Attractive M&A Target

Merck Keytruda Gets Thumbs Up From Adcom For Bladder Cancer

Merck & Co., Inc. MRK said the Oncologic Drugs Advisory Committee of the FDA voted 9 to 4 in favor of recommending the company's Keytruda for the treatment of certain patients with high-risk, non-muscle invasive bladder cancer.

The company expects a PDUFA action date of January 2020, based on priority review.

Rigel Chief Commercial Officer Quits

Rigel Pharmaceuticals, Inc. RIGL said its chief commercial officer Eldon Mayer has resigned, effective Dec. 23, to pursue an opportunity with an emerging company. The company said it has begun a search for a replacement, and until a new person assumes office, the commercial leadership team will directly report to CEO Raul Rodriguez.

Offerings

Neoluekin priced its previously announced underwritten public offering of 8.925 million shares of its common stock at $8.40 per share. The company expects to generate gross proceeds of about $75 million from the offering. The offering is expected to close on or about Dec. 20.

On The Radar

Adcom Meeting

FDA's Oncologic Drugs Advisory Committee will discuss Oncologic Drugs Advisory Committee Epizyme Inc's EPZM NDA for tazemetostat tablets for treating patients with metastatic or locally advanced epithelioid sarcoma not eligible for curative surgery. The briefing document released Monday showed that the FDA, though commending the company for exploring tazemetostat as a potential therapy for epithelioid sarcoma in a biologically rational way, expressed concerns about insufficient evidence to conclude that the investigational drug confers benefits in patients.