The Daily Biotech Pulse: AbbVie's Migraine Drug OK'd By FDA, Amicus Spins Off Gene Therapy Business, Geovax In-Licenses Cancer Drug

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

Click here for accessing Benzinga's FDA Calendar

Scaling The Peaks

(Biotech Stocks Hitting 52-week Highs Sept. 28)

Acceleron Pharma Inc. XLRN
Alkermes plc ALKS
Apyx Medical Corporation APYX
Avadel Pharmaceuticals plc AVDL
BELLUS Health Inc. BLU
BriaCell Therapeutics Corp. BCTX
Fennec Pharmaceuticals Inc. FENC
Innoviva, Inc. INVA

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Sept. 28)

4D pharma plc LBPS
Akoya Biosciences, Inc. AKYA
Aziyo Biologics, Inc. AZYO
BioVie Inc. BIVI
Burning Rock Biotech Limited BNR
Centessa Pharmaceuticals Limited CNTA
Emergent BioSolutions Inc. EBS
First Wave BioPharma, Inc. FWBI
Galapagos NV GLPG
Galecto, Inc. GLTO
GBS Inc. GBS
Graybug Vision, Inc. GRAY (released full-data analysis from the 18-month Phase 2b trial of GB-102 for the treatment of wet age-related macular degeneration)
HCW Biologics Inc. HCWB
Humanigen, Inc. HGEN (announced the European Medicines Agency has appointed a rapporteur and a co-rapporteur as part of the pre-submission process related to its plan to submit an MAA for the use of lenzilumab to treat patients hospitalized with COVID-19)
InMed Pharmaceuticals Inc. INM
Ionis Pharmaceuticals, Inc. IONS
Kintara Therapeutics, Inc. KTRA
Lantern Pharma Inc. LTRN (announced a collaboration to leverage artificial intelligence to identify eligible patient subgroups faster and with more accuracy in a Phase 2 study of never-smokers with non-small cell lung cancer)
MaxCyte, Inc. MXCT
Nautilus Biotechnology, Inc. NAUT
NeuroPace, Inc. NPCE
Passage Bio, Inc. PASG (announced additional preclinical data from its fibroblast activation protein-alpha inhibitor program called PNT2004)
Pharming Group N.V. PHAR
Pluristem Therapeutics Inc. PSTI
POINT Biopharma Global Inc. PNT
Poseida Therapeutics, Inc. PSTX
RenovoRx, Inc. RNXT
Scopus BioPharma Inc. SCPS
Sight Sciences, Inc. SGHT
Spruce Biosciences, Inc. SPRB
TCR2 Therapeutics Inc. TCRR
Titan Pharmaceuticals, Inc. TTNP
Tiziana Life Sciences PLC TLSA
Vertex Pharmaceuticals Incorporated VRTX
XBiotech Inc. XBIT
Zynex, Inc. ZYXI

Stocks In Focus

AbbVie's Oral Migraine Preventative Drug Gets FDA Approval

AbbVie, Inc. ABBV announced that the U.S. Food and Drug Administration approved Qulipta (atogepant) for the preventive treatment of episodic migraine in adults. Qulipta is the first and only oral calcitonin gene-related peptide receptor antagonist specifically developed for the preventive treatment of migraine, the company said.

The stock was up 0.68% to $108.07 in premarket trading.

Lilly's Erbitux Gets Label Expansion For Use In Colorectal Cancer Patients With Certain Mutation

Eli Lilly & Company LLY said the FDA has granted approval of a new indication for Erbitux, in combination with Braftovi, marketed by Pfizer, Inc. PFE, for the treatment of adult patients with metastatic colorectal cancer with a BRAF V600E mutation, as detected by an FDA-approved test, after prior therapy.

With this approval, Erbitux has now received seven FDA approvals to treat certain types of CRC and squamous cell carcinoma of the head and neck.

Lilly shares were up 1.42% to $224.75 in premarket trading.

Addex Commences Phase 2a Study of Dipraglurant For Treating Involuntary Contractions of Eyelid Muscles

Addex Therapeutics Ltd ADXN announced the initiation of a Phase 2a clinical study with dipraglurant as a potential treatment for blepharospasm, a type of dystonia characterized by involuntary contractions or spasms of the eyelid muscles resulting in sustained eyelid closure, which can result in substantial visual disturbance or functional blindness.

Dipraglurant selectively targets the metabotropic glutamate receptor subtype 5 to downregulate neurotransmission through allosteric modulation.

The stock was seen 1.91% higher at $9.63 in premarket trading.

Pfizer Announces Positive Phase 3 Data For Pneumococcal 20-valent Conjugate Vaccine

Pfizer announced positive top-line results from a Phase 3 study evaluating the safety and immunogenicity of Prevnar 20 (Pneumococcal 20-valent Conjugate Vaccine) in adults 65 years of age or older when administered at the same time as the seasonal influenza vaccine.

Responses elicited by PREVNAR 20 for all 20 serotypes and by seasonal influenza vaccine when given together were non-inferior to those elicited by the vaccines when administered one month apart. The safety profile of Prevnar 20 was similar when the vaccines were co-administered as compared to when each vaccine was administered separately, one month apart.

"This study adds to the body of evidence further supporting that pneumococcal conjugate vaccines may be co-administered with influenza vaccines, this time studied with the adjuvanted influenza vaccine," the company said.

Pfizer shares were adding 0.46% to $43.24 in premarket trading.

Fortress' Caelum To Be Acquired by AstraZeneca

Fortress Biotech, Inc. FBIO said AstraZeneca AZN's Alexion unit has exercised its option to acquire Caelum, a company founded by Fortress.

Caelum is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases

Amicus to Spin-off Gene Therapy Business Through a SPAC Deal

Amicus Therapeutics, Inc. FOLD announced its intent to launch a next-generation genetic medicine company, Caritas Therapeutics, Inc., through a definitive business combination agreement pursuant to which the Amicus gene therapy business will be acquired by ARYA Sciences Acquisition Corp IV ARYD, a SPAC, sponsored by Perceptive Advisors.

The transaction will result in two independent publicly traded companies with attractive stand-alone investment profiles. Amicus will become the largest shareholder in Caritas with about 36% ownership stake and retain co-development and commercialization rights to the Fabry and Pompe gene therapy programs as well as negotiation rights on select future muscular dystrophy programs.

Upon consummation of the business combination, current Amicus CEO John Crowley will lead Caritas as chairman and CEO and will become the Chairman Emeritus and chief strategic advisor for Amicus. Current Amicus COO Bradley Campbell will be named as CEO of Amicus.

Amicus shares were up 8.55% to $11.05 in premarket trading.

Celldex Presents Positive Early-stage Data For Skin Inflammation Treatment

Celldex Therapeutics, Inc. CLDX announced positive data on measurements of symptom control and quality of life from its ongoing Phase 1b clinical trial of CDX-0159 in patients with antihistamine refractory cold urticaria and symptomatic dermographism, the two most common forms of chronic inducible urticaria.

A single dose of CDX-0159 (3 mg/kg) resulted in a rapid and sustained improvement in urticaria control and greatly reduced disease impact on quality of life, as measured by the Urticaria Control Test and Dermatology Life Quality Index, the company said.

The data was presented at the European Association of Dermatology and Venereology 30th Anniversary Congress.

Nanobiotix Announces Publication of Positive Preclinical Data For Radio-enhancer Therapy In Treating Tumors

Nanobiotix S.A. NBTX announced the publication in the International Journal of Radiation Oncology, Biology, Physics, preclinical findings supporting further exploration of radio-enhancer NBTXR3 as a new therapeutic option seeking to induce significant tumor cell death when activated by radiotherapy and prime immune response, while also overcoming resistance to anti-PD-1.

Catalyst Biosciences' Lead Drug Gets Orphan Drug Designation For Treating Factor VII Deficiency

Catalyst Biosciences, Inc. CBIO said the FDA has granted orphan drug disease designation for its lead product candidate, subcutaneous Marzeptacog alfa for the treatment of factor VII deficiency.

Scholar Rock's Cancer Drug Issued Patent With an Expiry of May 2040

Scholar Rock Holding Corporation SRRK said the U.S. Patent and Trademark Office issued U.S. Patent No. 11,130,803, with an expiry of May 2040, including 313 days of patent term adjustment.

This US patent includes composition-of-matter claims, providing product protection for SRK-181, an investigational clinical candidate being developed to overcome primary resistance to and increase the number of patients who could benefit from checkpoint inhibitors, such as anti-PD-(L)1 therapies.

SRK-181 is being evaluated in the two-part DRAGON trial in patients with locally advanced or metastatic solid tumors exhibiting primary resistance to anti-PD-(L)1 therapy.

Geovax Labs In-licenses Immuno-oncology Asset Being Evaluated For Solid Tumors

GeoVax Labs, Inc. GOVX said it has entered into an assignment and license agreement with PNP Therapeutics, that grants GeoVax exclusive rights to develop and commercialize Gedeptin, a novel patented product for the treatment of solid tumors.

A Phase 1/2 trial, evaluating the safety and efficacy of repeat cycles of Gedeptin therapy in patients with recurrent head and neck squamous cell carcinoma, with tumors accessible for injection and no curable treatment options, is currently enrolling.

The stock was jumping 31.82% to $5.51 in premarket trading.

NRx's Zyesami Found to Be Safe In Phase 3 Study In Severe or Critical COVID-19 Patients

NRx Pharmaceuticals, Inc. (NRXP) provided a safety update on Zyesami, which is being tested in a Phase 3 study sponsored by the NIH. In its second scheduled analysis, the study's independent Data Safety Monitoring Board found no new safety concerns, after reviewing a total of 231 patients, and recommended continued enrollment.

The study represents one of three ongoing studies of Zyesami in severe or critical COVID-19 patients.

The stock was advancing 5.48% to $10.01 in premarket trading.

On The Radar

PDUFA Dates

The FDA is scheduled to rule on Mirum Pharmaceuticals, Inc. MIRM's new drug application for maralixibat as a treatment option for cholestatic pruritus in patients with Alagille syndrome. ALGS is a rare liver disease for which there are currently no approved therapies.

Clinical Readouts/Presentations

International Symposium on Retinal Degeneration Presentations

Applied Genetic Technologies Corporation AGTC: 12-month findings from two Phase 1/2 studies of subretinal gene therapy for achromatopsia in adults and low-dose pediatrics (9:35 am) and 12-month analysis of macular structure using optical coherence tomography from a Phase 1/2 study of subretinal gene therapy drug AGTC-501 for X-linked retinitis pigmentosa (10:35 am)

Editas Medicine, Inc. EDIT: initial clinical data from the BRILLIANCE clinical trial of EDIT-101 for the treatment of Leber congenital amaurosis 10, a CEP290-related retinal degenerative disorder.

Oligonucleotide Therapeutics Society Presentations

Scopus BioPharma Inc. SCPS: Phase 1 data from the DUET-01 study of bispecific oligonucleotide targeting TLR9 and STAT3 signaling for B cell non-Hodgkin lymphoma immunotherapy

IPOs

LumiraDx Limited, a next-gen point of care diagnostic testing company and CA Healthcare Acquisition Corp. CAHC, a SPAC traded on Nasdaq announced the completion of the previously announced merger. Trading will begin on Nasdaq under the ticker symbol "LMDX" for the common shares and "LMDXW" for the warrants assumed by LumiraDx from CAHC.

Market News and Data brought to you by Benzinga APIs