The Daily Biotech Pulse: ASH Presentations, Axsome's Positive Midstage Readout, Follow-On Offerings

The Daily Biotech Pulse: ASH Presentations, Axsome's Positive Midstage Readout, Follow-On Offerings

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 Dec. 7)

  • Affimed NV AFMD (reacted to ASH presentation)
  • Amicus Therapeutics, Inc. FOLD
  • Allovir Inc ALVR (moved on ASH presentation)
  • Atara Biotherapeutics Inc ATRA -announced a collaboration with Bayer AG BAYRY for CAR T-cell therapies for solid tumors
  • Avanos Medical Inc AVNS
  • Beam Therapeutics Inc BEAM (moved on ASH presentation)
  • Bioanalytical Systems, Inc. BASI
  • BioCryst Pharmaceuticals, Inc. BCRX - announced $325 million in funding from Royalty Pharma plc RPRX and Athyrium Capital 
  • BioLife Solutions Inc BLFS
  • BridgeBio Pharma Inc BBIO (announced joint venture with Maze Therapeutics for precision medicines to treat cardiovascular disease)
  • CareDx Inc CDNA
  • Castle Biosciences Inc CSTL (announced publication of clinical results for its skin cancer detection test)
  • Cellectis SA CLLS
  • Cerevel Therapeutics Holdings Inc CERE
  • Chimerix Inc CMRX (announced FDA acceptance of NDA for brincidofovir as a medical countermeasure for smallpox)
  • Codiak BioSciences Inc CDAK
  • Crispr Therapeutics AG CRSP (moved on ASH presentation)
  • CureVac BV CVAC
  • Denali Therapeutics Inc DNLI
  • Editas Medicine Inc EDIT (reacted to ASH presentation)
  • Eidos Therapeutics Inc EIDX
  • Fate Therapeutics Inc FATE (reacted to ASH presentation)
  • Foghorn Therapeutics Inc. FHTX
  • Frequency Therapeutics Inc (NASDAQ: FREQ
  • Gamida Cell Ltd GMDA (moved on ASH presentation)
  • Guardant Health Inc GH (moved on presentation at the San Antonio Breast Cancer Symposium)
  • Halozyme Therapeutics, Inc. HALO
  • Inhibrx Inc INBX
  • Intellia Therapeutics Inc NTLA (reacted to ASH presentation)
  • Inspire Medical Systems Inc INSP )
  • Iovance Biotherapeutics Inc IOVA
  • Kura Oncology Inc KURA (moved on ASH presentation)
  • Masimo Corporation MASI
  • Merrimack Pharmaceuticals Inc MACK
  • NeoGenomics, Inc. NEO
  • NGM Biopharmaceuticals Inc NGM
  • Novocure Ltd NVCR
  • Nurix Therapeutics Inc NRIX (moved on ASH presentation)
  • Obalon Therapeutics Inc OBLN
  • Ocular Therapeutix Inc OCUL
  • Organogenesis Holdings Inc ORGO
  • Pacific Biosciences of California Inc PACB
  • PMV Pharmaceuticals Inc PMVP
  • PTC Therapeutics, Inc. PTCT
  • Repare Therapeutics Inc RPTX
  • Sesen Bio Inc SESN (announced a commercialization partnership with Chinese CDMO Qilu for supply of its investigational oncology asset vicineum)
  • Spero Therapeutics Inc SPRO
  • Syndax Pharmaceuticals Inc SNDX (reacted to ASH presentation)
  • Tcr2 Therapeutics Inc TCRR
  • Travere Therapeutics Inc TVTX
  • Twist Bioscience Corp TWST
  • Ultragenyx Pharmaceutical Inc RARE (presented data from the Phase 1/2 study of GTX-102 in Angelman syndrome)
  • Veracyte Inc VCYT
  • XOMA Corp XOMA

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Dec. 7)

  • Lyra Therapeutics Inc LYRA (reacted to a mid-Phase 2 readout of its LYR-210 in treating chronic rhinosinusitis with or without nasal polyps)
  • Maravai Lifesciences Holdings Inc MRVI

Stocks In Focus

ASH Presentations

Agios Pharmaceuticals Inc AGIO announced updated data from the Phase 1 study evaluating mitapivat in adults with sickle cell disease showing, as of the data cut-off, six of 11 efficacy evaluable patients (55%) achieved a hemoglobin increase of more than1 g/dL from baseline, all at doses of 50 mg or less.

The company also noted dose-dependent increases in ATP and decreases in 2,3-DPG, as well as decreases in total bilirubin, LDH and absolute reticulocyte count. Safety profile was also consistent with previous studies, it added.

Agios said it expects to initiate a pivotal program in 2021.

Trillium Therapeutics Inc TRIL presented at the ASH conference with updated data for TTI-622, which is being evaluated as a monotherapy in patients with advanced relapsed or refractory lymphoma. Updated data as of a Nov. 3 cut-off date showed one patient at the 12 mg/kg dose level achieved a stable disease assessment.

The company also said dosing of TTI-621 — another pipeline asset evaluated in a Phase 1 dose escalation study in relapsed or refractory hematologic malignancies — of up to 0.5 mg/kg conducted under dose-limiting toxicity criteria is complete.

The stock was down 7.18% at $14.22 premarket Tuesday. 

Uniqure NV QURE announced two-year follow-up data from the Phase 2b study of Etranacogene Dezaparvovec, a AAV5-Padua hFIX variant and codenamed AMT-061, in adults with severe or moderate-severe hemophilia B treated irrespective of pre-existing anti-capsid neutralizing antibodies, showing all three patients have sustained FIX activity at therapeutic levels after a one-time administration.

Mean FIX activity for the three patients at two years after administration was 44.2% of normal compared to 41% of normal at 52 weeks.

The company said in a five-year follow-up Phase 1/2 study that AMT-060 gene therapy showed stable FIX expression and durable reductions in bleeding and FIX consumption.

The stock was down 4.54% to $41.80 in after-hours trading.

Axsome Reports Positive Phase 2 Results For AXS-05 In Major Depressive Disorder

Axsome Therapeutics Inc AXSM announced positive results from the open-label Phase 2 COMET-SI trial of AXS-05 in patients with major depressive disorder who have suicidal ideation.

Patients treated with AXS-05 experienced rapid reduction of suicidal ideation, rapid functional improvement and rapid, substantial and durable improvements in overall depressive symptoms, the company said.

AXS-05 was well-tolerated in the COMET trial, it added.

BeiGene's In-Licensed Leukemia Drug Approved In China

Beigene Ltd BGNE said the China National Medical Products Administration, or NMPA, has approved Blincyto for injection for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. The biologics license application was submitted by Amgen, Inc. AMGN and received priority review by the Center for Drug Evaluation of the NMPA. Amgen had licensed the drug to BeiGene under a strategic collaboration.

Takeda's Swelling Disorder Drug Approved In China

Takeda Pharmaceutical Co Ltd TAK said China's NMPA has approved Takhzyro subcutaneous injection for prophylaxis to prevent attacks of hereditary angioedema in patients 12 years and older.

"This milestone demonstrates Takeda's ongoing commitment to support the HAE community globally, as we work to expand access to TAKHZYRO and potentially 14 more highly innovative Takeda medicines to China's patients over the next five years," said Sean Shan, president of Takeda China.

Related Link: Week Ahead In Biotech: FDA To Decide On Emergency Use Of Pfizer's COVID Vaccine, Hematology Conference Presentations Pick Up Pace

Qiagen Raises Full-Year Guidance, Sounds Out Strong Performance In 2021

Molecular diagnostics company Qiagen NV QGEN raised its full-year 2020 net sales and adjusted earnings per share growth guidance, and also guided to strong performance in 2021.

The company expects net sale growth of at least 32% for the fourth quarter and adjusted EPS of 64-65 cents in constant exchange rate. For the full-year 2020, the company expects net sales growth of 22% and adjusted EPS of $2.13-$2.14.

Offerings

Marinus Pharmaceuticals Inc MRNS announced an underwritten public offering of its common stock. All of the shares to be sold in the offering will be offered by the company.

The stock was down 9.14% premarket at $14.21.

Arcturus Therapeutics Holdings Inc ARCT priced an underwritten public offering of 1.365 million shares of its common stock at $110 each, for raising gross proceeds of $150 million.

The closing of the offering is expected to occur on or about Dec. 1.

The stock was down 5.79% premarket at $116.50. 

Repligen Corporation RGEN priced its underwritten public offering of 1.5 million shares at $181 per share. The offering is expected to close on or about Dec. 10, and will likely raise $271.5 million in gross proceeds.

The stock was down 0.6% to $184.80 premarket. 

Syndax Pharma said it plans to offer, subject to market and other conditions, shares of its common stock in an underwritten public offering. All the shares are to be sold by the company.

Atara announced that it has commenced an underwritten public offering of $150 million in shares of its common stock. All of the shares in the proposed offering will be sold by the company.

The stock was down 1.3% premarket at $25.89.

Sutro Biopharma Inc STRO said it intends to offer 5 million shares of its common stock in a underwritten public offering. All of the shares of common stock are being offered by the company.

The stock ended down 3.65% to $21.10 in after-hours trading.

On The Radar

Clinical Readouts

Harpoon Therapeutics Inc HARP is scheduled to present a clinical update for its three most advanced TriTAC programs — namely HPN424 for the treatment of metastatic castration resistant prostate cancer, HPN536 initially in development for ovarian and pancreatic cancer and mesothelioma and HPN217 for relapsed/refractory multiple myeloma.

Evelo Biosciences Inc EVLO will present at the San Antonio Breast Cancer Symposium with a poster of additional data from a Phase 1/2 trial of EDP1503 in triple-negative breast cancer.

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

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