The Daily Biotech Pulse: Gamida Gains On Positive Regulatory Development, Orphazyme & ADMA Preannouncements, ImmunityBio & Leap Present Positive Data

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

Stocks In Focus

Orphazyme Preannounces Positive FY21 Results

Orphazyme A/S ORPH announced an upward revision to its 2021 net revenue guidance from 30 million Danish Krone to 40 million Danish Krone ($4.6 million - $6.1 million) to 35 million Danish Krone to 37 million Danish Krone ($5.3 million-$5.6 million). The company also narrowed its operating loss outlook.

The company noted that the improved financial outlook for 2021 is mainly driven by increased visibility on net revenues from the use of arimoclomol in the Early Access Program in France and lower-than-expected operating expenses due to strict cost management.

The stock was surging up 7.66% to $ 2.39 in premarket trading.

Galapagos' Filgotinib Gets U.K. Regulatory Nod For Treating Ulcerative Colitis

Galapagos NV GLPG announced that the Medicines and Healthcare products Regulatory Agency has granted a marketing authorization for Jyseleca (filgotinib 200mg tablets), as a new treatment for ulcerative colitis in Great Britain.

The MHRA has licensed an additional indication for Jyseleca for use in adult patients with moderately to severely active UC who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent.

Click here to access Benzinga's FDA Calendar

OncoCyte Announces Development And Co-Marketing Agreement For Cancer Diagnostics

OncoCyte Corporation OCX announced a development and co-marketing agreement for two distributed in vitro diagnostic assays on Thermo Fisher Scientific Inc.'s TMO Ion Torrent Genexus System. The agreement also grants Oncocyte rights to develop future companion diagnostics on the Genexus System.

Under the terms of the collaboration, Oncocyte will clinically validate Thermo Fisher's existing Oncomine Comprehensive Assay Plus on the Genexus System, paving the way toward IVD clearance for use in tumor profiling and future submissions as a companion diagnostic. Oncocyte will also develop its 27-gene expression DetermaIO test as a distributed kit on the Genexus. DetermaIO may predict response to immuno-oncology therapies based on data demonstrating potential pan-cancer utility and improvement over current standard-of-care tests.

OncoCyte shares were rising 4.11% to $2.28 in premarket trading.

Revance Says It Now Has Clear Path For Frown Line Treatment Resubmission Following Receipt Of Minutes Of Type A Meeting

Revance Therapeutics, Inc. RVNC announced the receipt of the official Type A meeting minutes from the U.S. Food and Drug Administration regarding the complete response letter received for DaxibotulinumtoxinA for Injection for glabellar lines. The Type A meeting was held on Dec. 15, 2021.

The FDA communication suggested that complete response requires qualification of new working cell bank, which the company said is in progress. The FDA also said a reinspection of its manufacturing facility will be necessary prior to approval.

"We believe we have a clear path to resubmission of the BLA for DaxibotulinumtoxinA for Injection for glabellar lines following our Type A meeting with the FDA," said Mark Foley, CEO of Revance.

In premarket trading, the stock was adding 1.33% to $14.50.

STRATA Skin Announces Deal to Distribute XTRAC Skin Disorder Treatment Lasers In Israel

STRATA Skin Sciences, Inc. SSKN said it has entered into an agreement with JuvenIL, the dermatology and aesthetics portion of Trimaco, an Israeli distributor for medical devices, to supply its XTRAC excimer lasers in Israel.

The stock was moving up 4% to $1.56 in premarket trading.

Nevro's Spinal Cord Stimulation System Gets Expanded Labeling For Treating Back Pain

Nevro Corp. NVRO announced receipt of FDA approval for expanded labeling for its Senza spinal cord stimulation system for the treatment of non-surgical refractory back pain. This approval is specific to Nevro's proprietary 10 kHz therapy and differentiates Nevro's Senza system as the only SCS system with specific labeling to treat NSRBP patients.

Gamida Cell To Initiate Rolling Regulatory Submission Of Application For Stem Cell Therapy For Blood Cancer Patients Needing Stem Cell Transplantation

Gamida Cell Ltd.GMDA announced that following receipt of positive Type B meeting correspondence from the FDA. The company said it plans to initiate a rolling biologics license application submission for omidubicel, a potentially life-saving treatment for patients with blood cancers in need of stem cell transplant.

Gamida Cell and the FDA have now reached alignment that analytical comparability has been established between the commercial manufacturing facility and the product that was manufactured for the Phase 3 study.

Based on this demonstration of comparability, along with the positive clinical results of the Phase 3 study, the FDA has agreed that the initiation of a rolling BLA submission is appropriate. Additional clinical data will not be required to initiate the BLA submission.

The stock was rising 21.17% to $2.69 in premarket trading.

Regeneron's Regulatory Application For Expanding Libtayo Label To Treat Lung Cancer Accepted For Review

Regeneron Pharmaceuticals, Inc. REGN announced that the FDA has accepted for review the supplemental BLA for Libtayo in combination with chemotherapy as first-line treatment in advanced non-small cell lung cancer. The target action date for the FDA decision is Sept. 19, 2022.

ADMA Preannounces Above-Consensus Q4, FY21 Revenues

ADMA Biologics, Inc. ADMA said it expects record fourth quarter 2021 revenues of approximately $26 million, compared to $14 million during the fourth quarter of 2020, reflecting an approximately 86% increase. For full year 2021, the company expects total revenues of approximately $81 million, compared to the year-ago revenues of $42 million.

Analysts, on average, estimate revenues of $21.76 million for the fourth quarter and $76.27 million for the year.

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

ASCO Gastrointestinal Cancers Symposium Presentations

ImmunityBio, Inc. IBRX announced interim results in its metastatic pancreatic cancer trial, showing that the overall survival rate for patients doubled compared to historical survival rate of three months after two prior lines of therapy. The data of the Phase 2 trial studying a combination immunotherapy also showed treatment-related serious adverse events were uncommon (8%) and no treatment-related deaths were reported. Based on these findings, ImmunityBio said it plans to meet with the FDA in 2022 to discuss the path for the approval of combination therapies for pancreatic cancer.

The stock was adding 11.47% to $6,90 in premarket trading.

Leap Therapeutics, Inc. LPTX said it will be presenting updated data from the DisTinGuish study, a Phase 2a clinical trial evaluating its anti-Dickkopf-1 antibody, DKN-01, in combination with tislelizumab, BeiGene Ltd.'s BGNE anti-PD-1 antibody, in patients with gastric or gastroesophageal junction cancer.

The new data is to demonstrate compelling efficacy from this combination regimen, driven by enhanced clinical responses and survival benefit.
Also to be presented are initial findings from the still-enrolling Part B of the clinical trial, studying DKN-01 and tislelizumab in second-line advanced G/GEJ patients with high tumoral DKK1 expression, showing the treatment is well tolerated with encouraging objective responses observed.

The stock was rallying 11.03% to $3.02 in premarket trading.

On The Radar

Clinical Readouts/Presentations

ASLAN Pharmaceuticals Limited ASLN is due to present at the Winter Clinical Dermatology Conference results from the dose escalation portion of the completed Phase 1b Proof-of- Concept study of ASLAN004 in atopic dermatitis

Cardiff Oncology, Inc. CRDF is schedule to present at the ASCO GI Cancers Symposium, updated data from its lead clinical program in KRAS-mutated metastatic colorectal cancer; Ahead of the presentation, the company is scheduled to host a conference call and webcast Tuesday, at 5 pm, to discuss the data.

Posted In: BiotechNewsPenny StocksGuidanceSmall CapFDAGeneral