The Daily Biotech Pulse: Orphazyme Plunges On Regulatory Setback In Europe, Pulmonx Earnings Disappoint, Amicus Declines To Spin Off Genetic Medicine Company

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

Stocks In Focus

Adagio Appoints COO David Hering As Interim CEO

Adagio Therapeutics, Inc., ADGI announced that its board has appointed David Hering as interim chief executive officer, effective immediately. Hering's appointment came following the resignation of Tillman Gerngross as CEO. The interim CEO will retain his role as chief operating officer.

Adagio also said It has initiated a formal CEO search and plan to appoint an executive with proven experience in bringing products to market and scaling operations.

Orphazyme Says EMA Committee Leaning Against Approval Of Arimoclomol

Orphazyme A/S ORPH said it has been notified by the European Medicines Agency's Committee for Medicinal Products for Human Use of a negative trend vote on marketing authorization application for its investigational product candidate, arimoclomol, for the treatment of Niemann-Pick disease type C.

The trend vote indicates that the CHMP's current orientation is to not approve arimoclomol when it convenes by the end of March 2022, Orphazyme said. In the light of the development and its financial situation, it said it will now assess its strategic options and provide an update to the market at the applicable time.

The U.S. Food and Drug Administration had rejected Orpahzyme's new drug application for arimoclomol in June 2021.

The stock was plunging 22.28% to $1.57 in premarket trading.

Grifols Announces R&D Collaboration For Antithrombin III Therapy In Sepsis

Grifols NASDAQ: GRFS) announced a collaboration with Endpoint Health, Inc., a precision medicine company, to develop and commercialize an Antithrombin III therapy to treat sepsis.

As part of the collaboration, and subject to the FDA's acceptance of a proposed investigational new drug and investigational device exemption filings, Endpoint Health will deploy its AI technology and its proprietary diagnostic test in a phase 2 trial designed to identify septic patients mostly likely to respond to AT-III. Upon FDA approval, the companies will invest in a build-out of AT-III production at Grifols sites.

Related Link: The Week Ahead In Biotech (Feb. 20-26): Reata Awaits FDA Decision, Bausch Health, Moderna Lead Earnings News Flow

Amicus Terminates SPAC Deal to Spinoff Genetic Medicine Company

ARYA Sciences Acquisition Corp IV ARYD, a SPAC sponsored by Perceptive Advisors, announced that, due to existing market conditions, it has mutually agreed with Amicus Therapeutics, Inc. FOLD to terminate their previously announced business combination agreement, effective immediately.

The proposed spin-off of the genetic medicine company in a SPAC deal valued at $600 million was announced in September.

G1 Therapeutics' Chinese Partner Reports Positive Late-Stage Data For Trilaciclib To Mitigate Chemo Effect In Lung Cancer Patients

G1 Therapeutics, Inc.'s GTHX Chinese partner Simcere Pharmaceutical announced that the phase 3 clinical registration study of Trilaciclib in patients with extensive-stage small cell lung cancer has met its primary endpoint.

The drug is being co-developed in China by Simcere and G1 Therapeutics. The latest data has shown a significantly decrease in duration of severe neutropenia of patients receiving chemotherapy in Cycle 1. Trilaciclib is an anti-tumor drug with comprehensive myeloprotective effect that can reduce chemotherapy-induced myelosuppression.

It was approved in the U.S. in February 2021.

Fulgent Announces Up to $40M Investment In Spatial Genomics

Fulgent Genetics, Inc. FLGT announced a strategic investment in Spatial Genomics, a developer of sequential fluorescence in situ hybridization technology.

Fulgent is investing up to $40 million to lead Spatial Genomics' Series A financing, which totals approximately $56 million. The company said it is also entering into commercial arrangements with Spatial to integrate Spatial Genomics' seqFISH technology into Fulgent's comprehensive genomic testing platform.

Separately, the company reported fourth-quarter revenues of $251.7 million and non-GAAP income of $3.48 per share. The company expects revenues of $245 million for the first quarter and $600 million for the fiscal year 2022. The results and guidance were above consensus expectations.

The stock was sliding 10.49% to $53.82 in premarket trading.

Click here to access Benzinga's FDA Calendar.

Earnings
Pulmonx Corporation LUNG reported fourth-quarter revenues of $13.7 million, up 39% year-over-year. The net loss per share widened from 27 cents to 35 cents. The results were in line with estimates.

The company expects revenues of $55 million to $60 million for the full-year 2022 and $9 million to $10 million for the first quarter, with both metrics trailing the consensus estimates by a wide margin.

The stock was slipping 21.87% to $20.51 in premarket trading.

TransMedics Group, Inc.'s TMDX fourth-quarter net revenue climbed 27% year-over-year to $9.7 million, ahead of the consensus estimate of $7.52 million. The net loss per share widened from 23 cents to 46 cents, which was also wider than the consensus loss estimate of 42 cents per share.

The company guided 2022 revenues to $49 million to $55 million, below the average analyst estimate of $62.12 million.

The stock was slipping 9.54% to $11.38 in premarket trading.

Vanda Pharmaceuticals, Inc. VNDA reported fourth-quarter net product sales of $68 million, a 1% increase from a year ago. The net income per share fell from 15 cents to 12 cents. For 2022, the company expects net product sales of $240 million to $280 million. Analysts, on average, estimate revenues of $313.62 million for the quarter.

In premarket trading, the stock was moving down 10.94% to $11.64.

BioMarin Pharmaceutical Inc.'s BMRN fourth-quarter revenues fell 1% to $449.8 million. The company reversed to a loss of 32 cents per share from a profit of 12 cents per share. The bottom-line result was in line with the consensus. The company guided 2022 revenues to $2.05 billion to $2.15 billion, roughly in line with the $2.14-billion consensus estimate.

In premarket trading, the stock was slipping 9.11% to $75.

On The Radar

Clinical Readouts/Presentations

Clene Inc. CLNN will present updated blinded data from its VISIONARY-MS Phase 2 study of CNM-Au8 for the treatment of chronic optic neuropathy and results from its REPAIR-MS Phase 2 trial of CNM-Au8 in the stable relapsing MS population. The presentations will be in the form posters at the Americas Committee for Treatment and Research in Multiple Sclerosis being held in West Palm Beach, Florida.

Otonomy, Inc. OTIC is scheduled to present at the Annual Scientific and Technology Meeting of the American Auditory Society with data from the Phase 1/2 study of eurotrophin OTO-413 for hearing loss

Earnings

• Agios Pharmaceuticals, Inc. AGIO (before the market open)
• Editas Medicine, Inc. EDIT (before the market open)
• Moderna, Inc. MRNA (before the market open)
• Pacira BioSciences, Inc. PCRX (before the market open)
• Sage Therapeutics, Inc. SAGE (before the market open)
• Vericel Corporation VCEL (before the market open)
• AbCellera Biologics Inc. ABCL (after the market close)
• CareDx, Inc CDNA (after the market close)
• Axonics, Inc. AXNX (after the market close)
• Collegium Pharmaceutical, Inc. COLL (after the market close)
• Emergent BioSolutions Inc. EBS (after the market close)
• Codexis, Inc. CDXS (after the market close)
• ICU Medical, Inc. . ICUI (after the market close)
• Invitae Corporation NVTA (after the market close)
• Iovance Biotherapeutics, Inc. IOVA (after the market close)
• Merit Medical Systems, Inc. MMSI (after the market close)
• LeMaitre Vascular, Inc. LMAT (after the market close)
• MacroGenics, Inc. MGNX (after the market close)
• Natera, Inc. NTRA (after the market close)
• OPKO Health, Inc. OPK (after the market close)
• Sangamo Therapeutics, Inc. SGMO (after the market close)
• Silk Road Medical, Inc SILK (after the market close)
• Travere Therapeutics, Inc. TVTX (after the market close)
• Y-mAbs Therapeutics, Inc. YMAB
• Vir Biotechnology, Inc. VIR (after the market close)

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