The Daily Biotech Pulse: European Regulatory Nod For Xeris, Amicus Falls On Data, 4 IPOs, FDA Decision For Mallinckrodt's StrataGraft Delayed

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 Feb. 12)

• 4D Molecular Therapeutics Inc FDMT
• Adagene Inc ADAG (went public Tuesday)
• Agios Pharmaceuticals Inc AGIO
• AnaptysBio Inc ANAB (received a double upgrade from JPMorgan)
• AngioDynamics, Inc. ANGO
• Applied Molecular Transport Inc. AMTI
• argenx SE – ADR ARGX
• Atea Pharmaceuticals Inc AVIR
• AtriCure Inc. ATRC
• Ayala Pharmaceuticals Inc AYLA
• Bausch Health Companies Inc BHC
• Bolt Biotherapeutics Inc BOLT
• C4 Therapeutics Inc CCCC
• Cara Therapeutics Inc CARA
• Celcuity Inc CELC
• Checkpoint Therapeutics Inc CKPT
• Collplant Biotechnologies Ltd – ADR CLGN
• DermTech Inc DMTK (announced award of contract for coverage of its skin cancer gene expression test)
• ESSA Pharma Inc EPIX (reacted to first-quarter results)
• Evogene Ltd EVGN
• FibroGen Inc FGEN
• G1 Therapeutics Inc GTHX
• Genetron Holdings Ltd – ADR GTH
• GenMark Diagnostics, Inc GNMK (moved on M&A speculation)
• Guardant Health Inc GH
• Guardion Health Sciences Inc GHSI
• Horizon Therapeutics PLC HZNP
• Immunome Inc IMNM
• Infinity Pharmaceuticals Inc. INFI (announced mid-Phase 2 data for eganelisib in combination of Opdivo for treating advanced urothelial cancer)
• Intra-Cellular Therapies Inc ITCI
• Lucira Health Inc LHDX (went public Feb. 5)
• Medpace Holdings Inc MEDP
• Merit Medical Systems, Inc. MMSI
• Merrimack Pharmaceuticals Inc MACK
• Monopar Therapeutics Inc MNPR (publication of a peer-reviewed study showing the potential utility of MNPR-101 as a uPAR imaging agent to improve surgical outcomes in bladder cancer)
• NeoGenomics, Inc. NEO
• Novo Nordisk A/S NVO
• Oncternal Therapeutics Inc ONCT
• Organovo Holdings Inc ONVO
• Pacific Biosciences of California Inc PACB (reacted to fourth-quarter results)
• Pacira Biosciences Inc PCRX
• PAVmed Inc PAVM
• PLx Pharma Inc PLXP
• Prothena Corporation PLC PRTA (announced fourth-quarter results)
• Seelos Therapeutics Inc SEEL
• Syneos Health Inc SYNH
• Vanda Pharmaceuticals Inc. VNDA (reacted to fourth-quarter results)
• Vericel Corp VCEL

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Feb. 12)

• Galapagos ADR Representing Ord Shs GLPG
• Vallon Pharmaceuticals, Inc. VLON (went public Wednesday)

Stocks In Focus

NantKwest Gets FDA Nod For Expanding Early Stage Study Of COVID-19 Vaccine

NantKwest Inc NK and ImmunityBio announced the receipt of FDA authorization to expand Phase I testing of a bivalent hAd5 T-cell COVID-19 vaccine.

The FDA authorized the expansion of a currently active, multi-cohort trial of the subcutaneous version of the vaccine in order to study the addition of sublingual boosts.

The FDA also authorized a second Phase I study that will examine the addition of an oral boost to the subcutaneous prime administration.

As a result, the companies will enroll another 105 participants in the U.S. trials of the vaccine candidate, which is unique in targeting both the spike (S) and nucleocapsid (N) proteins of the SARS-CoV-2 virus, the companies said.

NantKwest shares were up 14.06% at $29.70 premarket Friday.

Pompe Disease Combo Treatment From Amicus Flunks Late-Stage Study

Amicus Therapeutics, Inc. FOLD announced topline results from its Phase 3 PROPEL pivotal trial for AT-GAA (cipaglucosidase alfa and miglustat), its investigational two-component therapy for the treatment of late-onset Pompe disease.

The primary endpoint, while numerically greater, did not achieve statistical significance for superiority in the AT-GAA arm as compared to the alglucosidase alfa arm.

In premarket trading, the stock was down 23.65% to $14.30. 

Mesoblast Reports Positive Late-Stage Results For Stem Cell Therapy To Treat Chronic Back Pain

Mesoblast limited MESO announced positive results from the Phase 3 trial of its allogeneic mesenchymal precursor cell therapy rexlemestrocel-L in 404 enrolled patients with chronic low back pain due to degenerative disc disease refractory to conventional treatments.

The results indicate that a single injection of rexlemestrocel-L may provide a safe, durable and effective opioid-sparing therapy for patients with chronic inflammatory back pain due to degenerative disc disease, and the greatest benefits are seen when the drug is administered earlier in the disease process before irreversible fibrosis of the intervertebral disc has occurred, the company said. 

The stock was down 5.39% premarket to $10.36. 

Bausch Rallies After Icahn Discloses New Stake

Shares of Bausch rose after a 13-D filing by Icahn Capital revealed a 7.8% stake in the company, with the purchase premised on the assumption that the shares are undervalued. The activist investor suggested he plans to discuss with the management and board initiatives to enhance shareholder, including evaluation of strategic options.

The stock rallied 6.28% to $30 in after-hours trading.

Related Link: The Week Ahead In Biotech (Feb. 7-13): Regeneron FDA Decision, Earnings, IPOs and Conference Presentations In The Spotlight

Mallinckrodt Says FDA Decision On Burn Treatment Delayed As COVID-19 Holds Up Site Inspection

Mallinckrodt PLC MNKKQ said the FDA has informed the company that it is deferring action, pending a site inspection, on the Stratatech biologics license application for StrataGraft, an investigational allogeneic cellularized scaffold product in development for the treatment of adult patients with deep partial-thickness burns.

This is due to COVID-19-related travel restrictions, which are delaying a required manufacturing site inspection.

Xeris Gets European Regulatory Nod For Glucagon Pen To Treat Low Blood Sugar Levels

Xeris Pharmaceuticals Inc XERS announced that the European Commission has approved Ogluo injection for the treatment of severe hypoglycemia in adults, adolescents and children aged 2 years and over with diabetes mellitus. The marketing authorization is valid in all 27 countries in the European Union, plus Iceland, Norway and Liechtenstein.

The stock was advancing 27.45% to $8.45 in premarket trading Friday.

Alkermes Gains On Insider Buying

Alkermes Plc ALKS shares gained after SEC filings revealed two of its directors purchased shares in the company by exercising stock options and restricted stock unit award.

The stock was 5% higher premarket at $21.02. 

Clearpoint Neuro Announces Multiproduct Development Agreement For Neurosurgery Market

Clearpoint Neuro Inc CLPT announced a multiproduct joint development and option agreement with Blackrock Microsystems, which specializes in neuroscience, neural engineering and the neural prosthetics space. Blackrock electrodes are used in brain-computer-interfaces to restore function for patients suffering from spinal cord injuries, neurological disorders and amyotrophic lateral sclerosis.

The companies expect initial product launches in 2023, starting with the Microelectric Recording platform and to be followed with offerings including smart biopsy needles and other implantable neural electrodes.

The stock was adding 10.06% to $28 premarket Friday. 

Earnings

Applied DNA Sciences Inc APDN reported fiscal-year first-quarter revenue of $1.6 million, up from $634,000 in the year-ago period. The net loss per share narrowed from $1.12 to 88 cents.

The stock was down 18.62% premarket at $10.23. 

Aytu Bioscience Inc AYTU reported a strong increase in second-quarter net revenus from $3.175 million in fiscal year 2020 to $15.147 million in fiscal year 2021. The net loss per share widened from 12 cents to 72 cents.

The stock was down 4.69% premarket at $8.33. 

Offerings

Corvus Pharmaceuticals Inc CRVS said it has commenced an underwritten public offering of shares of its common stock. All of the shares to be sold in the offering will be offered by the company.

The stock was down 8.63% premarket at $3.60. 

Diffusion Pharmaceuticals Inc DFFN announced an increase to the size of the previously announced public offering and purchase on a firm commitment basis of 29.268 million shares of common stock at a price of $1.025 per share.

The stock was down 18.66% premarket at $1.09. 

Protalix Biotherapeutics Inc PLX priced its previously announced underwritten public offering of 7.619 million shares of its common stock at a price of $4.60 per share. The offering is subject to customary closing conditions and is expected to close on Feb. 17

In premarket trading, the stock was down 16.93% at $4.81. 

BridgeBio Pharma Inc BBIO announced the launch of a secondary public offering of 3 million shares of its common stock by selling stockholder KKR Genetic Disorder L.P. The company clarified that it is not selling any shares and will not receive any of the proceeds of the offering.

The stock was down 3.37% premarket at $65. 

Phio Pharmaceuticals Corp PHIO said it has entered into definitive agreements with several health care-focused institutional investors for the purchase and sale of 2.247 million shares of its common stock, at a purchase price of $3.42 per share, in a registered direct offering priced at-the-market for gross proceeds of approximately $7.7 million.

The stock was moving down 3.6% premarket to $3.48. 

Infinity Pharmaceuticals Inc. INFI said it has commenced an underwritten public offering of its common stock. All of the shares in the offering will be sold by the company.

The stock rose 5.59% premarket to $4.72. 

Collplant said it has entered into definitive agreements with several health care-focused and other institutional investors for the sale of 2 million ADSs at a purchase price of $17.50 per ADS, in a registered direct offering. Each ADS represents one ordinary share of the company.

On The Radar

Clinical Readouts

17th Annual WORLDSymposium Presentations

Avrobio Inc AVRO: uppdated results from a phase 1/2 study of hematopoietic stem cell gene therapy for cystinosis and updated Phase 2 data for AVR-RD-01 in Fabry disease (Friday, Feb. 12)

Abeona Therapeutics Inc ABEO: updated results from Phase 1/2 study of ABO-101 gene therapy for Mucopolysaccharidosis IIIB and Phase 1/2 study of ABO-102 gene therapy for Mucopolysaccharidosis IIIA (Friday, Feb. 12)

Denali Therapeutics Inc DNLI: data from Phase 1/2 study of DNL310 in Mucopolysaccharidosis II patients

TCT Meeting Presentations

Equillium Inc EQ: interim data from the Phase 1b/2 EQUATE study of itolizumab in acute graft-versus-host disease

Takeda Pharmaceutical Co Ltd TAK: Phase 3 data for TAK-620 in the treatment of transplant recipients with refractory/resistant cytomegalovirus infections

Angiogenesis, Exudation, and Degeneration meeting Presentations

NGM Biopharmaceuticals Inc NGM: data from Phase 1 study of NGM621 in in geographic atrophy

Earnings

ImmunoGen, Inc. IMGN (before the market open)

IPOs

Longeveron LLC, a clinical stage biotech developing cellular therapies for specific aging-related and life-threatening conditions, priced its downsized initial public offering of 2.66 million shares at $10, below the estimated price range of $10-$12. The Miami, Florida-based company's shares will begin trading on the Nasdaq under the ticker symbol "LGVN."

Talis Biomedical Corporation, which develops molecular diagnostic tests for infectious diseases at the point-of-care, priced its IPO of 13.8 million shares of its common stock at $16 per share, at the upper end of the estimated price range of $14-$16. The gross proceeds to Talis from the offering are expected at about $220.8 million. The shares will begin trading on the Nasdaq under the ticker symbol "TLIS."

Decibel Therapeutics priced its IPO of 7.062 million shares of its common stock at a price of $18 per share compared to the estimated price range of $16-$18. All of the shares of common stock are being offered by Decibel. The shares of the clinical-stage biotech developing treatments to restore and improve hearing and balance is expected to begin trading on the Nasdaq under the ticker symbol "DBTX."

NexImmune, Inc., a clinical-stage biotech developing immunotherapy to employ the body's own T cells to generate immune response that mimics natural biology, priced its upsized IPO of 6.471 million shares of its common stock at $17 per share, at the high end of the estimated price range of $15-$17. The gross proceeds are expected to be about $110 million.

The shares will begin trading on the Nasdaq under the symbol "NEXI."

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