The Daily Biotech Pulse: FDA Rejects FibroGen's Anemia Drug, Crinetics Gains On Positive Readout, BioCryst Withdraws Public Offering

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. 10)

• Alnylam Pharmaceuticals, Inc. ALNY
• BioNTech SE BNTX
• Caribou Biosciences, Inc. CRBU (IPOed July 23)
• Connect Biopharma Holdings Limited CNTB
• Cortexyme, Inc. CRTX
• Erasca, Inc. ERAS (IPOed July 16)
• Fulcrum Therapeutics, Inc. FULC (reacted to a positive readout for its sickle cell disease candidate)
• Intersect ENT, Inc. XENT
• Ironwood Pharmaceuticals, Inc. IRWD
• Maravai LifeSciences Holdings, Inc. MRVI
• Moderna, Inc. MRNA
• Monte Rosa Therapeutics, Inc. GLUE
• Oramed Pharmaceuticals Inc. ORMP
• Pfizer Inc. PFE
• Protagonist Therapeutics, Inc. PTGX - announced resolution of contract dispute with Zealand Pharma A/S ZEAL
• Quest Diagnostics Incorporated DGX
• Rani Therapeutics Holdings, Inc. RANI (IPOed July 30)
• Repligen Corporation RGEN
• West Pharmaceutical Services, Inc. WST

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Aug. 10)

• ACADIA Pharmaceuticals Inc. ACAD
• Akero Therapeutics, Inc. AKRO
• Arena Pharmaceuticals, Inc. ARNA
• Atai Life Sciences N.V. ATAI
• AVROBIO, Inc. AVRO
• Axsome Therapeutics, Inc. AXSM
• Biomea Fusion, Inc. BMEA
• Biophytis SA BPTS
• bluebird bio, Inc. BLUE
• Cellectar Biosciences, Inc. CLRB
• CytomX Therapeutics, Inc. CTMX
• CymaBay Therapeutics, Inc. CBAY
• Edgewise Therapeutics, Inc. EWTX
• Ensysce Biosciences, Inc. ENSC
• Epizyme, Inc. EPZM
• Eton Pharmaceuticals, Inc. ETON
• Exicure, Inc. XCUR
• EyeGate Pharmaceuticals, Inc. EYEG
• Foghorn Therapeutics Inc. FHTX (announced its second-quarter results)
• Freeline Therapeutics Holdings plc FRLN
• Grifols, S.A. GRFS
• HOOKIPA Pharma Inc. HOOK
• Incyte Corporation INCY
• Karyopharm Therapeutics Inc. KPTI
• Kiniksa Pharmaceuticals, Ltd. KNSA
• Kura Oncology, Inc. KURA
• MediciNova, Inc. MNOV
• Nektar Therapeutics NKTR
• Neurocrine Biosciences, Inc. NBIX
• Otonomy, Inc. OTIC
• Outset Medical, Inc. OM
• Progenity, Inc. PROG
• Relmada Therapeutics, Inc. RLMD
• RxSight, Inc. RXST (IPOed July 30)
• Seres Therapeutics, Inc. MCRB
• Spruce Biosciences, Inc. SPRB
• Stoke Therapeutics, Inc. STOK (announced second-quarter results)
• Talis Biomedical Corporation TLIS
• Teligent, Inc. TLGT
• TFF Pharmaceuticals, Inc. TFFP
• Trillium Therapeutics Inc. TRIL
• Viveve Medical, Inc. VIVE
• ZIOPHARM Oncology, Inc. ZIOP (reacted to quarterly results)
• Zogenix, Inc. ZGNX

Stocks In Focus

Crinetics Announces Positive Phase 1 Data For Therapy To Treat Adenocorticotropic Hormone Excesses

Crinetics Pharmaceuticals, Inc. CRNX announced positive preliminary findings from the single ascending dose portion of a first-in-human Phase 1 clinical study, with CRN04894 demonstrating pharmacologic proof-of-concept for its investigational, oral, nonpeptide adrenocorticotropic hormone antagonist that is being developed for the treatment of conditions of ACTH excess, including Cushing's disease and congenital adrenal hyperplasia.

The reductions in cortisol observed in the study suggest that CRN04894 is bound with high affinity to its target receptor on the adrenal gland and blocking the activity of ACTH. CRN04894 was well tolerated and all adverse events were considered mild.

In premarket trading, the stock was gaining 7.95% to $19.

Point Biopharma Announces Early Completion Of Enrollment, Initial Dosing In Late-Stage Prostate Cancer Study

POINT Biopharma Global Inc. PNT announced the completion of enrollment and initial dosing of the 25-patient safety and dosimetry lead-in of its Phase 3 SPLASH study evaluating PNT2002 for the treatment of metastatic castration-resistant prostate cancer.

Achieve Life Sciences Announces Patents For Smoking Cessation Drug Dosing Regiment

Achieve Life Sciences, Inc. ACHV announced the U.S. patent office has issued two patents covering the novel 3 mg, three-times-a-day cytisinicline dosing regimen.

"Upon approval of cytisinicline in the United States, we expect these patents would be eligible for inclusion in the FDA's Orange Book and could provide us marketing exclusivity for the TID cytisinicline dosing regimen until at least the third quarter of 2040," the company said.

Second Sight Gets NIH Grant Supplement For Study of Implantable Medical Device Used To Provide Vision

Second Sight Medical Products, Inc. EYES said it has received a grant supplement from the NIH to fund a qualitative study to gather critical insights into how profoundly blind people weigh the risks and benefits of visual neuroprostheses.

The study will help the company better understand acceptable risks, necessary benefits and the appropriate risk/benefit balance for visual cortical prostheses from the perspective of the target population. The entirety of the $155,964 grant supplement will be provided to UCLA as a subcontractor to conduct the study.

The stock was up 2.1% at $3.89 in premarket trading.

Related Link: The Week Ahead In Biotech (Aug. 8-14): Jazz Pharma FDA Decision, Earnings Deluge and IPOs In The Spotlight

Qiagen, GT Molecular Offer Wastewater Workflow Solution For Surveillance Of COVID-19 Outbreaks

QIAGEN N.V. QGEN and U.S.-based GT Molecular said they have started to offer a complete wastewater workflow solution designed to enable surveillance of COVID-19 outbreaks by U.S. and Canadian laboratories, with future options to offer the solution in other countries.

FibroGen's Roxadustat NDA Shot Down With a Complete Response Letter

FibroGen, Inc. FGEN said the FDA has issued a complete response letter regarding the new drug application for roxadustat for the treatment of anemia of chronic kidney disease.

The FDA indicated that it will not approve the roxadustat NDA in its present form and has requested additional clinical study of roxadustat be conducted, prior to resubmission.

An Adcom that met in mid-July ruled against approving the drug.

FibroGen is co-developing the drug with AstraZeneca plc AZN. The drug has been approved in China, Japan, Chile and South Korea.

Akouos' Gene-Mediated Hearing Loss Drug Gets Orphan Drug Designation In Europe

Akouos, Inc. AKUS announced that the European Medicines Agency Committee for Orphan Medicinal Products issued a positive opinion on the company's application for orphan drug designation for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss.

The positive opinion was subsequently adopted by the European Commission.

BioCryst Cancels Plan To Offer Shares

BioCryst Pharmaceuticals, Inc. BCRX said it has withdrawn its proposed public offering.

"With our strong balance sheet, and increasing revenues from ORLADEYO® (berotralstat), we believe that current market conditions are not conducive to an offering on terms that would be in the best interests of our current stockholders," the company said.

The company said it believes its cash runway will last into 2023.

The stock was advancing 9.24% to $16.61 in premarket trading.

Offerings

Fulcrum, which reported positive early stage data Tuesday for an investigational therapy for inherited blood disorders, said it has commenced an underwritten public offering of $100 million in shares of its common stock.

All of the shares are being offered by Fulcrum.

The stock was slipping 0.91% to $18.60 in premarket trading.

Click here to access Benzinga's FDA Calendar

On The Radar

Earnings

• Haemonetics Corporation HAE (before the market open)
• OptiNose, Inc. OPTN (before the market open)
• Royalty Pharma plc RPRX (before the market open)
• IMV Inc. IMV (before the market open)
• Altimmune, Inc. ALT (before the market open)
• Gamida Cell Ltd. GMDA (before the market open)
• ADMA Biologics, Inc. ADMA (before the market open)
• TELA Bio, Inc. TELA (after the close)
• Navidea Biopharmaceuticals, Inc. NAVB (after the close)
• Evofem Biosciences, Inc. EVFM (after the close)
• Repro Med Systems, Inc. KRMD (after the close)
• Xenon Pharmaceuticals Inc. XENE (after the close)
• VolitionRx Limited VNRX (after the close)
• Cyclacel Pharmaceuticals, Inc. CYCC (after the close)
• MannKind Corporation MNKD (after the close)
• DiaMedica Therapeutics Inc. DMAC (after the close)
• Eyenovia, Inc. EYEN (after the close)
• GeoVax Labs, Inc. GOVX (after the close)
• Histogen Inc. HSTO (after the close)
• Revolution Medicines, Inc. RVMD (after the close)
• Panbela Therapeutics, Inc. PBLA (after the close)

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