The Daily Biotech Pulse: Leap, Phio, Silverback Among Early Movers On Oncology Conference Presentations, Aerie Slumps On Mixed Data, Axsome Commences Phase 3 Sleep Disorder Study

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 Sept. 15)

  • Alcon Inc. ALC
  • Bicycle Therapeutics plc BCYC
  • Bio-Techne Corporation TECH
  • Cytek Biosciences, Inc. CTKB
  • Enanta Pharmaceuticals, Inc. ENTA
  • IVERIC bio, Inc. ISEE
  • Kezar Life Sciences, Inc. KZR
  • Opiant Pharmaceuticals, Inc. OPNT
  • Repligen Corporation RGEN
  • Vera Therapeutics, Inc. VERA
  • Zentalis Pharmaceuticals, Inc. ZNTL

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Sept. 15)

  • 4D pharma plc LBPS (announced biomarker analyses from two studies of MRx0518 in solid tumor settings)
  • Achilles Therapeutics plc ACHL
  • Acutus Medical, Inc. AFIB
  • Aditxt, Inc. ADTX
  • Applied Molecular Transport Inc. AMTI
  • Ardelyx, Inc. ARDX
  • AzurRx BioPharma, Inc. AZRX
  • CVRx, Inc. CVRX
  • Dermata Therapeutics, Inc. DRMA
  • Eiger BioPharmaceuticals, Inc. EIGR
  • Forte Biosciences, Inc. FBRX
  • Gamida Cell Ltd. GMDA
  • Grifols, S.A. GRFS
  • MiMedx Group, Inc. MDXG
  • MorphoSys AG MOR
  • Nautilus Biotechnology, Inc. NAUT
  • NeuroPace, Inc. NPCE
  • Oyster Point Pharma, Inc. OYST
  • Sonoma Pharmaceuticals, Inc. SNOA
  • Spectrum Pharmaceuticals, Inc. SPPI
  • Spruce Biosciences, Inc. SPRB
  • Theravance Biopharma, Inc. TBPH (announced plans to reduce workforce by 75% amid focus on the company's respiratory drug portfolio and pipeline)
  • Titan Pharmaceuticals, Inc. TTNP
  • TScan Therapeutics, Inc. TCRX
  • Unity Biotechnology, Inc. UBX
  • Zynex, Inc. ZYXI

Stocks In Focus

Aerie Announces Mixed Results For Phase 2b Study Of AR-15512 In Dry Eye Disease

Aerie Pharmaceuticals, Inc. AERI reported topline results from a Phase 2b clinical study of AR-15512 ophthalmic solution for the treatment of patients with dry eye disease, showing greatest efficacy in the higher concentration, 0.003% BID, which Aerie said will be advanced to Phase 3 studies.

The study achieved statistical significance for multiple pre-specified symptom endpoints and multiple pre-specified sign endpoints. It also showed statistically significant improvement in both symptoms and signs as early as day 14 and continuous improvement through day 84. Both formulations of AR-15512 were safe and well-tolerated, the company said. 

The study did not achieve statistical significance at the pre-determined primary endpoints at day 28, according to Aerie. 

Aerie said it expects to have an end of Phase 2 meeting with the Food and Drug Administration in the first quarter of 2022. In order to complete development of AR-15512, the company plans to conduct two additional three-month Phase 3 efficacy studies and an additional safety study.

The stock was slumping 15.29% to $13.30 premarket Thursday. 

Travere Out-Licenses Rights To Kidney Disorder Drug In Europe, Australia, New Zealand To Vifor

Travere Therapeutics, Inc. TVTX and Vifor Pharma announced the companies have entered into a joint collaboration and licensing agreement for the commercialization of sparsentan in Europe, Australia and New Zealand.

Sparsentan is a novel investigational product candidate currently being evaluated in pivotal Phase 3 clinical studies for the treatment of focal segmental glomerulosclerosis and IgA nephropathy, two rare progressive kidney disorders and leading causes of end-stage kidney disease.

Under the terms of the agreement, Vifor Pharma will receive exclusive commercialization rights for sparsentan in Europe, Australia and New Zealand. Travere will receive an upfront payment of $55 million and be eligible for up to $135 million in payments tied to the achievement of certain regulatory and market access related milestones. Vifor Pharma will also make further payments in the form of sales milestones, and tiered double-digit royalties on net sales of sparsentan in Europe, Australia and New Zealand up to 40% at the high end of the royalty range.

Travere shares were down 5.44% to $22.25 in premarket trading.

Axsome Commences Late-Stage Study Of AXS-12 As Treatment Option For Chronic Sleep Disorder

Axsome Therapeutics, Inc. AXSM said it has enrolled the first patient in SYMPHONY, a Phase 3 study of AXS-12 in patients with narcolepsy. AXS-12 is a novel, oral, potent, and highly selective norepinephrine reuptake inhibitor. Topline results from the SYMPHONY trial are anticipated in the first half of 2023.

The stock was gaining 2.92% to $28.53 in premarket trading.

Related Link: The Week Ahead In Biotech (Sept. 12-18): Calliditas FDA Decision, Adcom Review For Pfizer/BioNTech COVID-19 Vaccine, Oncology Conference And IPOs

Thermo Fisher Companion Diagnostic Tests For Takeda's Lung Cancer Drug Approved By FDA

Thermo Fisher Scientific Inc. TMO's Oncomine Dx Target Test received FDA's premarket approval as a companion diagnostic to identify patients with epidermal growth factor receptor Exon20 insertion mutation-positive metastatic non-small cell lung cancer who are candidates for Exkivity, a targeted drug developed by Takeda Pharmaceutical Company Limited TAK.

Crinetics Reports Positive Early Stage Results For Congenital Hyperinsulism Drug

Crinetics Pharmaceuticals, Inc. CRNX announced positive preliminary findings from the single ascending dose cohorts of a first-in-human Phase 1 clinical study with CRN04777, demonstrating pharmacologic proof-of-concept for treating congenital hyperinsulinism.


NRx Pharmaceuticals, Inc. NRXP filed a prospectus for the resale from time to time of up to 2.93 million shares of common stock by certain selling securityholders under private placement and 8,545 shares of common stock by certain service providers.
The prospectus also related to the issuance of up to 2.86 million shares of common stock upon the exercise of preferred investment options and placement agent preferred investment options.

The stock was slipping 0.86% to $11.48 in premarket trading.

aTyr Pharma, Inc. LIFE announced that it has commenced an underwritten public offering of shares of its common stock. All of the shares to be sold in the offering are to be sold by aTyr.

The shares were losing 4.58% to $8.13 in premarket trading.

Click here to access Benzinga's FDA Calendar.

On The Radar

Clinical Readouts/Presentations

European Society for Medical Oncology 2021 Congress Presentations

Leap Therapeutics, Inc. LPTX announced updated positive data from the first-line cohort of the DisTinGuish Phase 2a clinical trial evaluating its anti-Dickkopf-1 antibody, DKN-01, in combination with BeiGene Ltd. BGNE's tislelizumab, in patients with gastric or gastroesophageal junction cancer.

The combo as first-line therapy was well-tolerated with compelling activity.

Leap shares were jumping 41.45% to $2.73 in premarket trading.

Silverback Therapeutics, Inc. SBTX presented interim clinical results from a Phase 1/1b clinical study of SBT6050 as a monotherapy and in combination with Merck & Co, Inc.MRK's Keytruda in patients with advanced or metastatic HER2-expressing or amplified solid tumors.

As a monotherapy and in combination with pembrolizumab, SBT6050 was generally well-tolerated, with an adverse event profile that is consistent with immune system activation and considered on-mechanism.

The stock was down 13.85% at $14 in premarket trading.

Phio Pharmaceuticals Corp. PHIO announced results of a new study showing that local treatment in vivo with INTASYL can cure tumors and generate systemic tumor immunity that is both durable and tumor-specific.

The results show that up to 83% of the animals treated with PH-3861 had a complete response when treated at low doses, namely doses suboptimal for monotherapy. Moreover, this treatment induced a durable and specific systemic anti-tumor immune response, without requiring further treatment.

The stock was advancing 10.66% to $2.18 in premarket trading.

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

Posted In: BiotechNewsPenny StocksOfferingsSmall CapFDAGeneral