The Daily Biotech Pulse: Mesoblast Slumps On FDA Rejection, AstraZeneca's Breakthrough Therapy Designation, 3 IPOs

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 Oct. 1)

• 10X Genomics Inc TXG
• AMAG Pharmaceuticals, Inc. AMAG ( moved on M&A speculation)
• Annexon Inc ANNX
• Axonics Modulation Technologies Inc AXNX
• Beigene Ltd BGNE
• BioLife Solutions Inc BLFS (closed SciSafe buy)
• Blueprint Medicines Corp BPMC
• Brainstorm Cell Therapeutics Inc BCLI
• CareDx Inc CDNA (announced Medicare reimbursement for AlloSure Heart, a donor-derived cell-free DNA test)
• CymaBay Therapeutics Inc CBAY
• Fate Therapeutics Inc FATE
• LifeSci Acquisition Corp LSAC
• Myokardia Inc MYOK
• Novocure Ltd NVCR
• Pacific Biosciences of California Inc PACB
• PPD Inc PPD
• Prelude Therapeutics Inc PRLD
• Qiagen NV QGEN
• Seattle Genetics, Inc. SGEN
• Shockwave Medical Inc SWAV
• Tcr2 Therapeutics Inc TCRR

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Oct. 1)

• Aduro BioTech Inc ADRO
• Alector Inc ALEC
• Dyne Therapeutics Inc DYN
• Gritstone Oncology Inc GRTS
• Lexicon Pharmaceuticals, Inc. (NASDAQ: LXRX
• Neos Therapeutics Inc NEOS
• Orchard Therapeutics PLC – ADR ORTX
• Outset Medical Inc OM
• PainReform Ltd PRFX
• Pandion Therapeutics Inc PAND
• Pliant Therapeutics Inc PLRX
• Predictive Oncology Inc POAI
• Recro Pharma Inc REPH
• SCYNEXIS Inc SCYX
• Silence Therapeutics ADR Representing 3 Ord Shs SLN
• Taysha Gene Therapies Inc TSHA (listed on Sept. 24)
• Theravance Biopharma Inc TBPH

Stocks In Focus

Mesoblast's Ryoncil Fumbles at Regulatory Hurdle

Mesoblast MESO said the FDA issued a complete response letter to its BLA for remestemcel-L for the treatment of pediatric steroid-refractory acute graft versus host disease. The FDA has sought the company conduct at least one additional study in adults and/or children to provide further evidence of the effectiveness of remestemcel-L for SR-aGVHD.

Mesoblast said it will urgently request a Type A meeting with the FDA, which could be held within 30 days, to discuss a potential accelerated approval, with a post-approval condition for an additional study.

The FDA also identified a need for further scientific rationale to demonstrate the relationship of potency measurements to the product's biologic activity, the company said.

The stock was on a T1 trading halt Thursday. In after-hours trading, the stock slumped 42.83% to $10.61.

Sanofi's Regulatory Application For Enzyme Replacement Therapy For Pome Disease Accepted For Review In Europe

Sanofi SA SNY said the EMA accepted for review the marketing authorization application for avalglucosidase alfa, as a long-term enzyme replacement therapy for the treatment of patients with Pompe disease. Avalglucosidase alfa is an investigational enzyme replacement therapy, which, if approved, would offer a potential new standard of care for patients with Pompe disease.

The company expects EU approval for the therapy in the second half of 2021.

AstraZeneca Farxiga Gets Breakthrough Therapy Designation

AstraZeneca plc AZN said its Farxiga has been granted breakthrough therapy designation in the U.S. for patients with chronic kidney disease, with and without type 2 diabetes.

Metacrine To Present Positive Proof-of-concept For NASH Drug

Metacrine Inc MTCR announced new pharmacokinetic and pharmacodynamic data from its MET409 Phase 1b proof-of-concept clinical trial in patients with non-alcoholic steatohepatitis, or NASH, showing robust and sustained FXR activation, which is expected to be key to optimizing therapeutic benefits.

"Combined with a favorable pharmacological and tolerability profile, these results further highlight the therapeutic potential of MET409 for patients with NASH," the company said.

The company is scheduled to present these findings as two posters at the American Association for the Study of Liver Diseases' The Liver Meeting Digital Experience scheduled between Nov. 13 and 15.

The stock rose 4.17% to $10.50 in after-hours trading.

Aduro's Shareholders Approve Merger Transaction With Chinook

Aduro BioTech Inc ADRO said at a special meeting of shareholders held Oct. 1, its shareholders approved its proposed merger with Chinook Therapeutics.

Following stockholder approval, the company announced a one-for-five reverse stock split. The shares will begin trading on a split-adjusted basis on Nasdaq, effective with the open of the market Friday.

The closing of the merger is anticipated to on or around Oct. 5. Following closing of the merger, the combined company will be renamed Chinook Therapeutics and trade under the trading symbol KDNY, Aduro said.

The stock was up 4.20% to $2.98 in after-hours trading.

Lab Corp. Gets EUA For New Tech to Improve Speed and Efficiency of PCR Test to Detect COVID-19

Laboratory Corp. of America Holdings LH said it received emergency use authorization from the FDA for a new, high-throughput method that uses heat and technology to extract RNA from samples collected for COVID-19 molecular testing. The advancement, the company said, will help improve the speed and efficiency of RT-PCR tests.

Clinical Readouts

Xencor Inc XNCR will present initial data from its Phase 1 dose-escalation study of tidutamab in patients with neuroendocrine tumors.

IPOs

Exton, Pennsylvania-based Immunome, Inc., which uses its proprietary human memory B cell platform to discover and develop antibody therapeutics, priced its upsized initial public offering, or IPO, of 3.25 million shares of its common stock at $12, at the middle of the estimated price range of $11-$13. The offering is expected to raise gross proceeds of $39 million. The shares will begin trading on the Nasdaq under the ticker symbol IMNM.

Cancer immunotherapy company Oncorus, Inc. priced its 5.8-million-share IPO at $15 apiece, for raising gross proceeds of $87 million. The Cambridge, Massachusetts-based company had earlier estimated a price range of $14-$16. The shares will begin trading on the Nasdaq under the ticker symbol ONCR.

Watertown, Massachusetts-based C4 therapeutics, Inc. priced its upsized IPO of 9.6 million shares at $19, above the estimated price range of $16-$18. The biopharma that develops therapeutic candidates to destroy disease-causing proteins for the treatment of cancer, neurodegenerative conditions and other diseases, said it expects to raise gross proceeds of $182.4 million from the offering. The shares will commence trading on the Nasdaq under the ticker symbol CCCC.