The Daily Biotech Pulse: FDA Greenlights Merck's Heart Failure Drug, Histogen Hit With Clinical Hold, Aptorum Gets Nod For Commencing Human Study

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 Jan. 19)

• 4D Molecular Therapeutics Inc FDMT
• ABIOMED, Inc. ABMD
• AcelRx Pharmaceuticals Inc ACRX
• Aclaris Therapeutics Inc ACRS ( reacted to a positive mid-stage readout)
• Adaptive Biotechnologies Corp ADPT
• Akari Therapeutics PLC AKTX
• Aldeyra Therapeutics Inc ALDX
• Alkermes Plc ALKS
• Apyx Medical Corp APYX
• Arvinas Inc ARVN
• Beigene Ltd BGNE
• Bicycle Therapeutics PLC BCYC
• BIO-TECHNE Corp TECH
• BioCryst Pharmaceuticals, Inc. BCRX
• Biodesix Inc BDSX
• BioNano Genomics Inc BNGO
• BioVie Inc BIVI
• Burning Rock Biotech Ltd BNR
• Cara Therapeutics Inc CARA
• CareDx Inc CDNA
• CASI Pharmaceuticals Inc CASI
• Cassava Sciences Inc SAVA
• Castle Biosciences Inc CSTL
• Cellectis SA CLLS
• Celsius Holdings, Inc. CELH
• Clearside Biomedical Inc CLSD
• Clearpoint Neuro Inc CLPT
• Corcept Therapeutics Incorporated CORT
• Curis, Inc. CRIS
• Dare Bioscience Inc DARE
• DiaMedica Therapeutics Inc DMAC
• Eli Lilly And Co LLY (announced an oncology licensing deal and received a ratings upgrade from Mizuho)
• ESSA Pharma Inc EPIX
• Eton Pharmaceuticals Inc ETON
• Evelo Biosciences Inc EVLO
• Evogene Ltd EVGN
• Fulgent Genetics Inc FLGT
• Genetron Holdings Ltd – ADR GTH
• Glaukos Corp GKOS
• Gritstone Oncology Inc GRTS (announced plans to commence clinical study of COVID-19 vaccine candidate)
• Halozyme Therapeutics, Inc. HALO (reacted to FDA approval for partner Janssen's drug to treat newly diagnosed light chain amyloidosis)
• Harpoon Therapeutics Inc HARP
• Idera Pharmaceuticals Inc IDRA
• ImmunoGen, Inc. IMGN
• Inari Medical Inc NARI
• Infinity Pharmaceuticals Inc. INFI
• INmune Bio Inc INMB
• Insulet Corporation PODD
• Integra Lifesciences Holdings Corp IART
• Inventiva ADR Representing Ord Shs IVA
• iTeos Therapeutics Inc ITOS
• Jazz Pharmaceuticals PLC JAZZ
• Johnson & Johnson JNJ
• Kaleido Biosciences Inc KLDO
• LeMaitre Vascular Inc LMAT
• Ligand Pharmaceuticals Inc. LGND
• Merus NV MRUS (announced an oncology licensing deal with Lilly)
• Natera Inc NTRA
• Neuronetics Inc STIM
• Orphazyme A S ADR NORPH
• PRA Health Sciences Inc PRAH
• Precision BioSciences Inc DTIL (announced FDA acceptance of IND for commencing blood cancer study)
• Pulse Biosciences Inc PLSE
• Purple Biotech ADR PPBT
• Repligen Corporation RGEN
• Shockwave Medical Inc SWAV
• Stoke Therapeutics Inc STOK
• Supernus Pharmaceuticals Inc SUPN
• Surface Oncology Inc SURF
• Sutro Biopharma Inc STRO
• Syneos Health Inc SYNH
• Taysha Gene Therapies Inc TSHA (announced receipt of pediatric disease and orphan drug designations for TSHA-105, an AAV9-based gene for SLC13A5-related epilepsy)
• TFF Pharmaceuticals Inc TFFP
• Twist Bioscience Corp TWST (announced signing of two licensing agreements)
• United Therapeutics Corporation UTHR
• Vericel Corp VCEL
• Vistagen Therapeutics Inc VTGN
• Zai Lab Ltd – ADR ZLAB

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Jan. 19)

• Gracell Biotechnologies Inc GRCL (declined despite the Chinese regulatory nod for commencement of a blood cancer study)

• Stocks In Focus

Calliditas to Present Clinical Pipeline Update at R&D Day

Calliditas Therapeutics Adr Rep 2 Ord Shs CALT said it will reveal near-term clinical development plans for setanaxib in primary biliary cholangitis and oncology at the R&D day to be held Wednesday morning. The company said it plans to initiate a pivotal Phase 2/3 study in PBC in the second half of 2021 and a Phase 2 proof-of-concept study in head and neck cancer this year.

The company said it will also present select data from the recently concluded Part A of the Phase 3 study NeflgArd with lead candidate drug Nefecon for the treatment of immunoglobin A nephropathy, showing a 9.5% rate of discontinuation of study treatment and 3.5% rate of discontinuation from the study. No adverse clinical effects were seen with regards to weight gain, blood pressure or HbA1c, reflecting a safety profile.

FDA Imposes Clinical Hold On Histogen's Knee Cartilage Regeneration Therapy Study

Histogen Inc HSTO said the FDA has verbally notified the company it has additional questions about its investigational new drug application package for the planned Phase 1/2 clinical trial of HST-003, which is intended to evaluate the safety and efficacy of human extracellular matrix, implanted within microfracture interstices and the cartilage defect in the knee to regenerate hyaline cartilage in combination with a microfracture procedure.

Based upon the verbal communication with FDA, the company said the agency indicated that the clinical hold is due to pending chemical, manufacturing and control information that is required to complete their review.

The stock fell 16.11% to 99 cents in after-hours trading.

Aptorum Gets Clearance In Canada to Commence First-ever Clinical Study

Aptorum Group Ltd APM said its subsidiary has received clearance from the Health Canada regarding the clinical trial application to commence a Phase 1 study of ALS-4, an orally administered small molecule drug intended to treat infections caused by Staphylococcus aureus including MRSA.

"This milestone supports the transition of Aptorum Group to a clinical-stage company and reflects the potential of our scientific rigor and novel approach of our products," said Clark Cheng, chief medical officer, of the company.

The stock was rallying 19.50% to $3.80 in premarket trading Wednesday.

Related Link: The Week Ahead In Biotech: FDA To Issue Decisions On Merck And Aurinia Drug Applications

Merck's Heart Failure Drug Approved by the FDA

Merck & Co., Inc.'s MRK said the FDA has approved Verquo, a soluble guanylate cyclase stimulator, to reduce the risk of cardiovascular death and heart failure hospitalization following a hospitalization for heart failure or need for outpatient intravenous diuretics in adults with symptomatic chronic heart failure and ejection fraction less than 45%.

Verquo, 2.5 mg, 5 mg, and 10 mg tablets, is being jointly developed with Bayer AG BAYRY.

Bristol-Myers Squibb's Regulatory Application Seeking Label Expansion For Opdivo Accepted For Priority Review

Bristol-Myers Squibb Co BMY said the FDA has accepted its supplemental Biologics License Application sBLA for Opdivo for the treatment of patients with resected esophageal or gastroesophageal junction cancer in the adjuvant setting, after neoadjuvant chemoradiation therapy. The FDA granted the application priority review and assigned a PDUFA goal date of May 20.

Offerings

Syros Pharmaceuticals Inc SYRS said it has priced an underwritten public offering of 5.4 million shares of common stock at $14 per share, resulting in gross proceeds of about $75.6 million. The proceeds of the offering are expected to be used to fund the development of Syros' ongoing clinical and preclinical programs, and for working capital and other general corporate purposes. All shares are being offered by the company.

The stock was down 4.68% to $14.25 in after-hours trading.

Merus announced the launch of a proposed underwritten public offering of up to $60 million of its common shares. All of the common shares are being offered by the company.

The stock fell 6.11% to $23.20 in after-hours trading.

CytomX Therapeutics Inc CTMX said it has commenced an underwritten public offering of $75 million shares of its common stock. All of the shares are to be offered by CytomX.

In after-hours trading, the stock moved down 6.84% to $7.90.

AcelRx Pharma announced an underwritten public offering of its common stock. All shares to be sold in the offering will be sold by AcelRx, subject to customary closing conditions.

The stock lost 11.49% to $2.08 in after-hours trading.

BioLineRx ADR Representing 15 Ord Shs BLRX said it has increased the size of the previously announced offering of 12.5 million ADSs of the company at a price to the public of $2.40 per ADS. The offering is expected to fetch the company $30 million in gross proceeds.

The stock slumped 17.67% to $2.61 in after-hours trading.

BioNano announced that it plans to sell, subject to market and other conditions, shares of its common stock in an underwritten public offering.

The stock plunged 10.83% to $8.15 in after-hours trading.

Chimerix Inc CMRX said it intends to sell, subject to market and other conditions, up to $75 million worth of shares of its common stock in an underwritten public offering.

In after-hours trading, the stock slid 0.62% to $8.05.

CareDxi said it intends to sell, subject to market and other conditions, up to $175 million of shares of its common stock in an underwritten public offering. All of the shares will be offered and sold by CareDx.

The stock edged down 0.41% to $92.95 in after-hours trading.

Passage Bio Inc PASG said it has commenced an underwritten public offering of 7 million shares of its common stock. All shares of common stock to be sold in the offering will be sold by the company.

The stock fell 1.89% to $28.50 in after-hours trading.

Editas Medicine Inc EDIT announced its intention to sell 3.5 million shares of its common stock in an underwritten public offering. All of the shares in the offering are to be sold by the company.

In after-hours trading, the stock was down 0.83% to $71.51.

On The Radar

Clinical Readouts:

Seres Therapeutics Inc MCRB will present at the Keystone Symposium Harnessing the Microbiome for Disease Prevention and Therapy, new data from its SER-109 Phase 3 study in recurrent C. difficile infection.

Related Link: Attention Biotech Investors: Mark Your Calendar For January PDUFA Dates