The Daily Biotech Pulse: Eton Snags Second FDA Nod For Month, Vaccine Updates from Moderna, CureVac

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. 29)

• Acceleron Pharma Inc XLRN (announced Health Canada approval of luspatercept for RBC transfusion-dependent anemia associated with beta-thalassemia)
• Adaptive Biotechnologies Corp ADPT
• Adial Pharmaceuticals Inc ADIL(announced EUA for antibody test for COVID-19)
• Arena Pharmaceuticals, Inc. ARNA
• Axonics Modulation Technologies Inc AXNX
• Beigene Ltd BGNE
• BioLife Solutions Inc BLFS
• Blueprint Medicines Corp BPMC
• Cardiff Oncology Inc CRDF
• Celldex Therapeutics, Inc. CLDX
• CymaBay Therapeutics Inc CBAY
• Fate Therapeutics Inc FATE
• Guardant Health Inc GH
• Inspire Medical Systems Inc INSP
• Insulet Corporation PODD
• Kaleido Biosciences Inc KLDO(announced dosing of first patient in ulcerative colitis study)
• Kura Oncology Inc KURA
• Lantern Pharma Inc. LTRN
• Momenta Pharmaceuticals, Inc. MNTA
• Natera Inc NTRA
• Novo Nordisk A/S NVO
• Pacific Biosciences of California Inc PACB
• Seattle Genetics, Inc. SGEN
• Shockwave Medical Inc SWAV
• Twist Bioscience Corp TWST

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Sept. 29)

• Alector Inc ALEC
• Dyne Therapeutics Inc DYN
• Greenwich Lifesciences Inc GLSI(debuted on Nasdaq Friday)
• Happiness Biotech Group Ltd HAPP
• Neos Therapeutics Inc NEOS
• Orchard Therapeutics PLC – ADR ORTX
• Outset Medical Inc OM
• PainReform Ltd PRFX (commenced preparations of pivotal Phase 3 study of PRF-110 for the treatment of post-operative pain)
• Renalytix AI PLC RNLX
• Satsuma Pharmaceuticals Inc STSA
• Teligent Inc (NEW JERSEY) TLGT
• Theravance Biopharma Inc TBPH
• Tricida Inc TCDA
• Uniqure NV QURE
• Vaccinex Inc VCNX

Stocks In Focus

Moderna's Coronavirus Vaccine Found Equally Effective Against At-Risk Older Population

Moderna Inc MRNA announced publication in the New England Journal of Medicine second interim results from the Phase 1 study of mRNA-1273 in older age cohorts, showing the investigational vaccine-induced consistently high levels of pseudovirus neutralization antibody titers in all participants in the 56-70 and 71+ age cohorts. It also elicited Th1-biased CD4 T cell responses in these age cohorts.

"Neutralizing antibody titers and T cell responses in the 56-70 and 71+ age cohorts were consistent with those reported in younger adults," the company said. The vaccine candidate was also well tolerated in all age cohorts.

"Given the increased morbidity and mortality of COVID-19 in older and elderly adults, these data give us optimism in demonstrating mRNA-1273's protection in this population, which is being evaluated in the Phase 3 COVE study," Moderna said.

Separately, Moderna's CDMO partner Lonza said new production lines at its Portsmouth, New Hampshire facility should start making vaccine ingredients in November, and three lines at its Swiss Alps facility will likely begin delivering by December, Reuters reported, citing company executives.

The stock rose 4.93% to $74 in after-hours trading.

CureVac Initiates Mid-stage Study of COVID-19 Vaccine

CureVac BV CVAC announced the dosing of the first participant in a Phase 2a clinical trial of its COVID-19 vaccine candidate, CVnCoV. The dose-confirmation study is being conducted in Peru and Panama and targets enrolling a total of 690 healthy participants in two distinct groups - older adults ages 61 and above, and younger participants 18 to 60 years old, the company said.

The company said it expects to report first comprehensive data from the Phase 2a study in older adults in the fourth quarter 2020.

In after-hours trading, the stock was up 8.12% to $49.39.

Eton's Hormone Replacement Therapy For Adrenocortical Insufficiency In Children Clears FDA Hurdle

Eton Pharmaceuticals Inc ETON said the FDA approved Alkindi Sprinkle, or hydrocortisone, oral granules as replacement therapy for adrenocortical insufficiency in children under 17 years of age.

Alkindi Sprinkle is the first and only FDA-approved granular hydrocortisone formulation for the treatment of adrenocortical insufficiency specifically designed for use in children, Eton said.

The company expects the drug to be commercially available in the fourth quarter of 2020.

The stock rallied 18.29% to $9.25 in after-hours trading.

CTI BioPharma To Begin Rolling NDA Submission For Drug to Treat Bone Marrow Cancer

CTI BioPharma Corp CTIC said it has reached an agreement with the FDA to submit an NDA for the potential accelerated approval of pacritinib as a treatment for myelofibrosis patients with severe thrombocytopenia. This follows the company's recent pre-NDA meeting with the agency.

The FDA has agreed to a rolling NDA submission which is expected to commence within a few weeks, with completion of the NDA submission anticipated in the first quarter of 2021, the company said.

The stock jumped 208.57% to $3.24 in after-hours trading.

Tonix's Fibromyalgia Study Extended to Include Additional Participants Following IDMC Review

Following a pre-planned interim analysis of a Phase 3 study of TNX-102 SL, 5.6 mg, for the management of fibromyalgia, Tonix Pharmaceuticals Holding Corp TNXP said the independent data monitoring committee made the non-binding recommendation that the trial continue to completion with the addition of 210 participants to the original sample size of 470 participants. This is the maximum number of participants that could be added under the interim statistical analysis plan, Tonix said.

The company now expects to report top-line results from the study in the fourth quarter.

The stock fell 6.67% to 84 cents in after-hours trading.

Pfizer Forges Oncology Partnership to Market Cancer Drug In Mainland China

Pfizer Inc. PFE and Hong Kong-based CStone Pharma announced the formation of a strategic collaboration for the development and commercialization of CStone's PD-L1 antibody sugemalimab, a cancer drug, in mainland China. The collaboration also envisages bringing additional oncology assets to the Greater China market. As part of the collaboration, Pfizer through its Hong Kong unit will make a $200 million equity investment in CStone.

CStone is also entitled to receive up to $280 million in milestone payments for sugemalimab and additional tiered royalties.
While Pfizer obtains exclusive commercialization rights in mainland China, CStone has the development and commercialization rights outside mainland China.

Catalyst Pharma To Appeal District Court Ruling In Lawsuit On LEMS Drug

Catalyst Pharmaceuticals Inc CPRX said following the federal judge upholding the verdict of the Magistrate Judge, granting summary judgment in favor of the FDA and Jacobus, and dismissing Catalyst's case, it intends to appeal the result to the Eleventh Circuit Court of Appeals.

The lawsuit filed by Catalyst challenged FDA decision to approve Jacobus' Ruzurgi for the treatment of Lambert-Eaton myasthenic syndrome, or LEMS, in patients 6 years to less than 17 years of age.

Catalyst's Firdapse was approved in 2018 for LEMS in adults.

The stock was down 2.49% to $3.13 in after-hours trading.

Iterum Plans to File NDA For Antibotic To Treat Uncomplicated Urinary Tract Infections

Iterum Therapeutics PLC ITRM said, based on discussions at a pre-NDA meeting with the FDA, it plans to proceed with an NDA submission for sulopenem etzadroxil/probenecid, a bilayer tablet, for the treatment of uncomplicated urinary tract infections in patients with a quinolone-resistant pathogen.

In premarket trading Wednesday, the stock was soaring 72.25% to $1.11.

Offerings

Aptorum Group Ltd APM, which rallied Tuesday on the setting up of a liquid biopsy diagnostics subsidiary, priced its offering of 2.769 million Class A ordinary shares and warrants to purchase up to another 2.769 million Class A Ordinary Shares, at a combined public offering price of $3.25 per share and related warrant.

The warrants have an exercise price of $3.25 per share are exercisable immediately upon issuance and expire on the five-year anniversary of the date of issuance, the company said.

Aptorum expects to raise gross proceeds of $9 million from the offering.

The stock was down 4.41% to $4.55 in after-hours trading.

Cardiff Oncology priced its previously announced underwritten public offering of 6.5 million shares of its common stock at $13.5 apiece. The gross proceeds from the offering are expected at $88 million.

The stock rose 6.42% to $14.91 in after-hours trading.

Arcutis Biotherapeutics Inc ARQT announced the commencement of an underwritten public offering of 4 million shares of its common stock. Concurrent with the public offering, the company said it plans to sell to OrbiMed Advisors, an affiliate of one of its directors, $35 million worth of shares in a private placement.

In after-hours trading, the stock slid 1.74% to $30.50.

PDUFA Date

Mesoblast limited MESO awaits FDA decision on its BLA for remestemcel-L (MSC-100-IV) in steroid-refractory acute graft versus host disease in children.