The Daily Biotech Pulse: AzurRx Jumps On Data, Agios' Regulatory Application For Mitapivat Accepted For Priority Review, FDA Nod For GlaxoSmithKline

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 Aug. 17)

• AbbVie Inc. ABBV
• Alkermes plc ALKS
• Century Therapeutics Inc IPSC
• Gilead Sciences, Inc. GILD
• GlaxoSmithKline plc GSK
• Edward Lifesciences Corp EW
• Eli Lilly and Company LLY (announced reorganization of business units)
• Innoviva Inc INVA
• Intuitive Surgical, Inc. ISRG
• Johnson & Johnson JNJ
• Maravai Lifesciences Holdings Inc MRVI
• NanoVibronix, Inc. NAOV
• Novo Nordisk A/S NVO
• Nuvalent Inc NUVL
• Omega Therapeutics Inc OMGA
• Pfizer, Inc. PFE
• Regeneron Pharmaceuticals Inc REGN (moved on news COVID-19-affected Texas Governor Greg Abbott is being treated with the company's antibody treatment)
• Quest Diagnostics Incorporated DGX
• ResMed Inc. RMD
• Virpax Pharmaceuticals Inc VRPX (moved on announcement regarding FDA response for pursuing MMS019 as a prophylactic treatment against SARS and influenza viruses for daily use as an OTC product)
• West Pharmaceutical Services, Inc. WST

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Aug. 17)

• 4D pharma plc LBPS
• Absci Corporation ABSI
• ABVC BioPharma, Inc. ABVC
• ACADIA Pharmaceuticals Inc. ACAD
• AcelRx Pharmaceuticals Inc ACRX (reacted to its second-quarter results)
• Achieve Life Sciences, Inc. ACHV
• Acurx Pharmaceuticals, Inc. ACXP (announced its second-quarter results)
• Adaptive Biotechnologies Corporation ADPT
• Adicet Bio Inc ACET
• Adverum Biotechnologies, Inc. ADVM
• ADMA Biologics Inc ADMA
• Agile Therapeutics, Inc. AGRX
• Akouos Inc AKUS
• Akoya Biosciences, Inc. AKYA
• Alzamend Neuro Inc ALZN
• Angion Biomedica Corp. ANGN
• Applied Genetic Technologies Corporation AGTC
• Aptinyx Inc. APTX
• Aptose Biosciences Inc. APTO
• Aravive, Inc. ARAV
• Arena Pharmaceuticals, Inc. ARNA
• Assertio Holdings, Inc. ASRT
• Athenex, Inc. ATNX
• Athira Pharma, Inc. ATHA (announced its second-quarter results)
• Avenue Therapeutics, Inc. ATXI
• AVITA Medical, Inc. RCEL
• Axsome Therapeutics, Inc. AXSM
• Aytu Biopharma, Inc. AYTU
• AzurRx BioPharma Inc AZRX
• Biocept Inc BIOC (reacted to its quarterly results)
• Biodesix, Inc. BDSX
• Biomea Fusion, Inc. BMEA
• BiondVax Pharmaceuticals Ltd. BVXV
• BioXcel Therapeutics Inc. BTAI
• Caladrius Biosciences, Inc. CLBS
• Candel Therapeutics Inc CADL (IPOed July 30)
• Catalyst Biosciences Inc CBIO
• Cellectis S.A. CLLS
• Chemomab Therapeutics Ltd. CMMB
• CNS Pharmaceuticals, Inc. CNSP
• Concert Pharmaceuticals Inc CNCE
• ContraFect Corporation CFRX
• Cue Biopharma Inc CUE
• CymaBay Therapeutics Inc CBAY
• CytomX Therapeutics Inc CTMX
• Deciphera Pharmaceuticals, Inc. DCPH
• Diffusion Pharmaceuticals Inc. DFFN
• DURECT Corporation DRRX
• Edgewise Therapeutics, Inc. EWTX
• Eloxx Pharmaceuticals, Inc. ELOX
• Ensysce Biosciences, Inc. ENSC (announced its second-quarter results)
• Enveric Biosciences, Inc. ENVB
• Epizyme, Inc. EPZM
• Esperion Therapeutics Inc ESPR
• Eton Pharmaceuticals, Inc. ETON (announced its second-quarter results)
• Evofem Biosciences, Inc. EVFM
• Exicure Inc XCUR
• FibroGen, Inc. FGEN
• Flexion Therapeutics, Inc. FLXN
• Forma Therapeutics Holdings Inc FMTX
• Frequency Therapeutics Inc FREQ
• Galecto, Inc. GLTO
• Gemini Therapeutics, Inc. GMTX
• Genocea Biosciences, Inc. GNCA
• GlycoMimetics, Inc. GLYC
• Heron Therapeutics, Inc. HRTX
• HOOKIPA Pharma Inc. HOOK
• Hoth Therapeutics, Inc. HOTH
• IMARA Inc. IMRA
• Indaptus Therapeutics, Inc. INDP
• InflaRx N.V. IFRX
• Insmed Incorporated INSM
• Intercept Pharmaceuticals Inc ICPT
• iRhythm Technologies Inc IRTC
• Itamar Medical Ltd. ITMR
• Kamada Ltd KMDA
• Karyopharm Therapeutics Inc. KPTI
• Keros Therapeutics, Inc. KROS
• Kiniksa Pharmaceuticals, Ltd. KNSA
• Kinnate Biopharma Inc. KNTE (announced its second-quarter results)
• Kura Oncology, Inc. KURA
• Liminal BioSciences Inc. LMNL (announced its second-quarter results)
• Lixte Biotechnology Holdings, Inc. LIXT
• Longeveron Inc. LGVN
• Madrigal Pharmaceuticals, Inc. MDGL
• Medicenna Therapeutics Corp. MDNA
• Moleculin Biotech, Inc. MBRX
• MorphoSys AG MOR
• Nabriva Therapeutics plc NBRV
• Nanobiotix S.A. NBTX
• Nektar Therapeutics NKTR
• Oncorus, Inc. ONCR
• OncoSec Medical Incorporated ONCS (announced management changes)
• Orchard Therapeutics plc ORTX
• Palisade Bio, Inc. PALI
• Panbela Therapeutics, Inc. PBLA
• Passage Bio, Inc. PASG
• Phio Pharmaceuticals Corp. PHIO
• Pluristem Therapeutics Inc. PSTI
• Praxis Precision Medicines, Inc. PRAX
• Progenity, Inc. PROG
• Protara Therapeutics, Inc. TARA
• Pulmatrix, Inc. PULM
• Qualigen Therapeutics, Inc. QLGN (announced its second-quarter results)
• Recursion Pharmaceuticals, Inc. RXRX
• Regencell Bioscience Holdings Limited RGC
• Rhythm Pharmaceuticals, Inc. RYTM
• Rockwell Medical, Inc. RMTI (announced its second-quarter results)
• RxSight, Inc. RXST (IPOed July 30)
• Scopus BioPharma Inc. SCPS
• scPharmaceuticals Inc. SCPH
• SI-BONE, Inc. SIBN
• Sigilon Therapeutics, Inc. SGTX
• Seres Therapeutics, Inc. MCRB
• Singular Genomics Systems, Inc. OMIC
• Soleno Therapeutics, Inc. SLNO
• Sonnet BioTherapeutics Holdings, Inc. SONN
• Sonoma Pharmaceuticals, Inc. SNOA (announced its second-quarter results)
• Stoke Therapeutics, Inc. STOK
• Syros Pharmaceuticals, Inc. SYRS
• T2 Biosystems, Inc. TTOO
• Taysha Gene Therapies, Inc. TSHA
• Teligent, Inc. TLGT
• TG Therapeutics, Inc. TGTX
• TherapeuticsMD, Inc. TXMD
• TransCode Therapeutics, Inc. RNAZ
• Unicycive Therapeutics, Inc. UNCY
• Universe Pharmaceuticals INC UPC
• VectivBio Holding AG VECT
• Viveve Medical, Inc. VIVE
• Vor Biopharma Inc. (VOR)
• VYNE Therapeutics Inc. VYNE
• X4 Pharmaceuticals, Inc. XFOR
• Xeris Pharmaceuticals, Inc. XERS
• ZIOPHARM Oncology, Inc. ZIOP
• ZIVO Bioscience, Inc. ZIVO

Stocks In Focus

Agios's Mitapivat Application Accepted For Regulatory Review

Agios Pharmaceuticals, Inc. AGIO said the Food and Drug Administration accepted its new drug application for mitapivat for the treatment of adults with pyruvate kinase deficiency. The NDA was granted a priority review designation and has been given a PDUFA action date of Feb. 17, 2022.

The stock was up 3.02% to $46 in after-hours trading.

Click here to access Benzinga's FDA Calendar

GlaxoSmithKline's Solid Tumor Drug Approved by FDA, Triggering Milestone Payment For AnaptysBio

GlaxoSmithKline announced that the FDA approved a second indication for Jemperli for the treatment of adult patients with mismatch repair deficient recurrent or advanced solid tumors, as determined by an FDA-approved test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options.

Jemperli was generated by AnaptysBio, Inc. ANAB using its proprietary somatic hypermutation antibody platform and subsequently developed by Tesaro, now a part of GSK, under a collaboration agreement.

AnaptysBio has earned a $20 million milestone payment as a result of this second FDA approval for Jemperli in dMMR recurrent or advanced solid tumors.

AnaptysBio was rallying 7.19% to $27.

AzurRx Reports Positive Phase 2 Data For Combo Therapy For Treating Pancreatic Insufficiency In Cystic Fibrosis Patients

AzurRx announced positive topline results from its Phase 2 combination trial evaluating MS1819 in combination with the current standard of care, porcine-derived pancreatic enzyme replacement therapy, for the treatment of severe exocrine pancreatic insufficiency in patients with cystic fibrosis.

"Data collected from 20 patients indicated that MS1819 in combination with PERT led to clinically meaningful improvements in the primary efficacy endpoint, the Coefficient of Fat Absorption," the company said.

The stock was jumping 38.09% to 72.8 cents in premarket trading.

Marinus Announces Positive Phase 2 Data For Oral Ganaxolone as Adjunctive Treatment For Seizures In Tuberous Sclerosis Complex Patients

Marinus Pharmaceuticals, Inc. MRNS announced topline data from its Phase 2 trial evaluating safety and efficacy of adjunctive oral ganaxolone treatment in 23 patients with seizures associated with tuberous sclerosis complex, with primary endpoint showing a median 16.6% reduction in 28-day seizure frequency relative to baseline in TSC patients.

The secondary endpoint of 50% responder rate at 30.4% is consistent with the Marigold Phase 3 CDKL5 deficiency disorder trial of 24.5 percent, the company said. Patients with focal seizures showed a median 25.2% reduction in focal seizure frequency, the most common seizure type in TSC patients, it added.

Marinus said it expects to begin enrollment in a global Phase 3 trial of adjunctive ganaxolone in approximately 160 TSC patients during the fourth quarter.

The stock was slipping 2.16% to $11.78 in premarket trading.

South Korea In Talks to Expedite Moderna COVID-19 Vaccine Supply

Moderna, Inc. MRNA is adding to Tuesday's gains. A report in the Korean Times said the nation's health officials along with Samsung Biologics are in advanced discussion with Moderna to expedite the latter's COVID-19 vaccine supply.

South Korea intends to receive 24 million doses from Moderna by the end of October. Moderna has a deal with South Korea to supply 40 million vaccine doses by the end of 2021, and out of this only 2.45 million doses have been delivered thus far.

Moderna shares were seen rising 1.79% to $409.05 in premarket trading.

Sutro's Antibody-drug Conjugate STRO-002 Gets Fast Track Designation For Treating Epithelial Ovarian, Fallopian Tube Cancer

Sutro Biopharma, Inc. STRO said the FDA has granted fast track designation for STRO-002, an antibody-drug conjugate, for the treatment of patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior lines of systemic therapy.

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

Cabaletta Announces Positive Safety Data From Phase 1 Study of DSG3-CAART In Pemphigus Vulgaris Patients

Cabaletta Bio, Inc. CABA announced positive 28-day data from the second dose cohort, at the 100 million cell dose level, in the Phase 1 clinical trial of DSG3-CAART for the treatment of patients with mucosal-dominant pemphigus vulgaris.

Despite a five-fold higher dose than the initial cohort, there were no clinically relevant adverse events or dose-limiting toxicities observed either acutely or in the 28-day DLT monitoring period following infusion, the company said.

The stock was up 3.10% at $7.64 in premarket trading.