The Daily Biotech Pulse: Sarepta Gene Therapy Study Readout, Novavax Vaccine Supply Deal, 2 IPOs

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 Jan. 7)

• AngioDynamics, Inc. ANGO (reacted to its fiscal-year 2021 second-quarter results)
• Apellis Pharmaceuticals Inc APLS
• Arvinas Inc ARVN
• Atea Pharmaceuticals, Inc. AVIR
• AxoGen, Inc Common Stock AXGN
• Axonics Modulation Technologies Inc AXNX
• BIO-TECHNE Corp TECH
• Bioanalytical Systems, Inc. BASI
• Codiak BioSciences Inc CDAK
• Crispr Therapeutics AG CRSP
• DermTech Inc DMTK
• ESSA Pharma Inc EPIX
• Evelo Biosciences Inc EVLO (announced expansion of clinical program and issued an update on key 2021 milestones)
• Fate Therapeutics Inc FATE
• Glaukos Corp GKOS
• Globus Medical Inc GMED
• Haemonetics Corporation HAE
• Halozyme Therapeutics, Inc. HALO
• Hologic, Inc. HOLX
• \I-Mab ADR IMAB
• Intellia Therapeutics Inc NTLA (the genomic editing company announced 2021 milestones and strategic priorities)
• Jazz Pharmaceuticals PLC JAZZ
• Johnson & Johnson JNJ
• Laboratory Corp. of America Holdings LH
• LeMaitre Vascular Inc LMAT
• Masimo Corporation MASI
• MiMedx Group Inc MDXG
• Neuronetics Inc STIM (announced expansion of its commercial organization)
• Oxford Immunotec Global PLC (NASDAQ: OXFD (announced a deal to be acquired by PerkinElmer,PKI)
• Pacific Biosciences of California Inc PACB
• Pacira Biosciences Inc PCRX(announced above-consensus preliminary revenues for 2020)
• PRA Health Sciences Inc PRAH
• Praxis Precision Medicines Inc PRAX (received rare pediatric disease and orphan drug designations for its severe pediatric epilepsy programs)
• PTC Therapeutics, Inc. PTCT
• Pulse Biosciences Inc PLSE
• Radius Health Inc RDUS
• Repare Therapeutics Inc RPTX
• Rhythm Pharmaceuticals Inc RYTM
• SAGE Therapeutics Inc SAGE (issued a corporate strategy update)
• Shockwave Medical Inc SWAV
• Solid Biosciences Inc SLDB
• United Therapeutics Corporation UTHR
• Vascular Biogenics Ltd VBLT
• Vericel Corp VCEL
• Vincera Pharma Inc VINC
• Vistagen Therapeutics Inc VTGN
• Xencor Inc XNCR (announced a cancer drug collaboration)
• Zai Lab Ltd – ADR ZLAB ( announced in-licensing of Chinese rights to argenx SE's – ADR ARGX efgartigimod)
• West Pharmaceutical Services Inc. WST

Down In The Dumps

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

• Fusion Pharmaceuticals Inc FUSN

Stocks In Focus

Sarepta Phase 2 Duchenne Muscular Dystrophy Gene Therapy Study Does Not Meet Primary Endpoint

Sarepta Therapeutics Inc SRPT announced topline results from Part 1 of the Phase 2 study, dubbed Study 102, that is evaluating its investigational gene therapy, SRP-9001, in Duchenne muscular dystrophy, showing the study did not achieve statistical significance on the primary functional endpoint of improvement in NSAA total score compared to placebo at 48 weeks post treatment.

The study, however, met the biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment, as measured by western blot, the company said. The company also noted that no new safety signals were identified, reinforcing the favorable safety profile observed to date.

The stock slumped 50.01% to $84.45 in after-hours trading.

In sympathy move, Solid Biosciences Inc SLDB fell 24.53% to $6.50 and Rocket Pharmaceuticals Inc RCKT declined a more modest 4.42% to $57.50.

Novavax Inks Deal With Australia to Supply Up to 61M Doses of Coronavirus Vaccine Candidate

Novavax, Inc. NVAX said it has executed an advance purchase agreement with Australia for 51 million doses of NVX-CoV2373, its COVID-19 vaccine candidate. This follows an agreement in principle that was announced in November 2020.

The company said it will work with Australia's regulatory agency, the Therapeutics Goods Administration, to obtain product approvals upon demonstrating efficacy in clinical studies. The company aims to deliver initial doses by mid-2021. The agreement also carries an option to purchase up to an additional 10 million doses. Additional terms of the agreement were not disclosed.

Merus' Cancer Drug Gets Fast Track Designation

Merus NV MRUS said the FDA has granted fast track designation to Zenocutuzumab for the treatment of patients with metastatic solid tumors harboring NRG1 gene fusions that have progressed on standard of care therapy.

The stock jumped 29.95% to $21 in after-hours trading.

Chimerix Buys Oncology Biotech Oncoceutics For Up to $438M

Chimerix Inc CMRX said it has acquired Oncoceutics, Inc., a privately-held, clinical-stage biotechnology company developing imipridones, a novel class of compounds, for $78 million, payable in cash and stock, subject to certain customary adjustments.

Oncoceutics shareholders will also potentially earn development, regulatory and sales milestones totaling up to $360 million across three development programs and royalties on combined sales of ONC201 and ONC206 of 15% up to $750 million in annual revenue and 20% above $750 million in annual revenue.

Oncoceutics' lead product candidate, ONC201, has been shown in clinical testing to selectively induce cell death in multiple cancer types. ONC201 is currently in a registrational clinical trial for recurrent H3 K27M-mutant glioma and a confirmatory response rate assessment is expected in 2021.

In premarket trading Friday, Chimerix shares were up 10.24% to $5.49.

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

Clearside Shares Rally On Insider Buying

Clearside Biomedical Inc CLSD shares rose after a filing with the SEC showed that company insider Bradford Whitmore purchased 350,750 shares of the company at a price of $2.851 apiece.

The stock was up 8.06% to $3.89 in after-hours trading.

Oncternal Announces Cell Therapy Collaboration Agreement With Swedish Academic Institution

Oncternal Therapeutics Inc ONCT announced a R&D collaboration with Karolinska Institutet in Stockholm, Sweden, to advance novel ROR1-targeting cell therapies focused on CAR-T cells and CAR-NK cells from the laboratory into the clinic.

In after-hours trading, the stock gained 3.16% to $4.89.

Offerings

Cerecor Inc CERC said it intends to offer shares, and, to a certain investor in lieu thereof, pre-funded warrants to purchase shares of common stock, in an underwritten public offering.

All of the securities will be offered by Cerecor.

The stock was down 6.19% to $2.73 in after-hours trading.

BioNano Genomics Inc BNGO said it plans to sell, subject to market and other conditions, shares of its common stock in an underwritten public offering. All of the shares in the proposed offering are to be sold by the company.

The stock shed 11.60% to $4.42 in after-hours trading.

On The Radar

IPOs

China-based Gracell Biotechnologies Inc, a clinical-stage oncology-focused cell therapy company, priced its initial public offering of 11 million ADSs, each representing five ordinary shares, at a price of $19.00 per ADS. The company had earlier estimated a price range of $16-$18. The gross proceeds from the offering are expected to be $209 million.

The ADSs have been approved for listing on the Nasdaq under the ticker symbol GRCL.

Cullinan Management, Inc., a biopharma focused on developing a diversified pipeline of targeted oncology and immuno-oncology therapies with transformative potential for cancer patients, priced its upsized initial public offering of 11. 9 million shares at $21 per share, for raising gross proceeds of $249.9 million. The Cambridge, Massachusetts-based company had previously planned to offer 10 million share, with the estimated price range at $19-$20.

The shares will begin trading on the Nasdaq under the ticker symbol CGEM.