The Daily Biotech Pulse: Kazia Jumps On Data Readout, Clinical Hold On Cellectis' Blood Cancer Study Lifted

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 Nov. 17)

• Aerpio Pharmaceuticals Inc ARPO
• ALX Oncology Holdings Inc ALXO (announced an oncology collaboration with Zymeworks Inc ZYME)
• Amicus Therapeutics, Inc. FOLD
• Atara Biotherapeutics Inc ATRA
• Biodesix Inc BDSX
• Biohaven Pharmaceutical Holding Co Ltd (NYSE: BHVN
• C4 Therapeutics Inc CCCC
• Champions Oncology Inc CSBR
• Clearpoint Neuro Inc CLPT
• Edwards Lifesciences Corp EW
• Generation Bio Co GBIO
• Halozyme Therapeutics, Inc. HALO
• Immunovant Inc IMVT
• Infinity Pharmaceuticals Inc. INFI
• Intellia Therapeutics Inc NTLA
• Insmed Incorporated INSM
• Keros Therapeutics Inc KROS
• Kymera Therapeutics Inc KYMR
• LeMaitre Vascular Inc LMAT
• NeoGenomics, Inc. NEO
• Neuronetics Inc STIM
• Outset Medical Inc OM
• Prelude Therapeutics Inc PRL
• PTC Therapeutics, Inc. PTCT(initiated Phase 1 trial of PTC518, which is being evaluated for Huntington's disease)
• Relay Therapeutics Inc RLAY
• Rocket Pharmaceuticals Inc RCKT
• Seres Therapeutics Inc MCRB
• Shockwave Medical Inc SWAV
• SpringWorks Therapeutics Inc SWTX
• SQZ Biotechnologies Co SQZ
• Sutro Biopharma Inc STRO
• Syndax Pharmaceuticals Inc SNDX
• Tcr2 Therapeutics Inc TCRR
• Trillium Therapeutics Inc TRIL
• Trinity Biotech plc TRIB (moved ahead of its quarterly results)
• Urovant Sciences Ltd UROV
• XOMA Corp XOMA

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Nov. 17)

• Freeline Therapeutics Holdings PLC FRLN

Stocks In Focus

Kazia To Present Positive Mid-stage Results For Brain Tumor Drug

Announcing summary of its scheduled presentation at the Society for Neuro-Oncology annual meeting, Kazia Therapeutics Ltd KZIA said new interim analysis of paxalisib phase 2 study in glioblastoma is highly consistent with prior data.

Median progression-free survival was 8.4 months compared to 5.3 months for the existing standard-of-care temozolomide, and median overall survival was also better at 17.5 months versus 12.7 months. Safety at 60mg dose showed profile similar to prior experience, with the most common toxicities including rash, mouth ulcers, and hyperglycemia, consistent with other PI3K and mTOR inhibitors.

The stock was jumping 19.13% to $11.77 in premarket trading Wednesday.

Sanofi Gets Fast Track Designation For Bleeding Disorder Drug, Priority Review For Pompe Disease Enzyme Replacement Therapy

Sanofi SA SNY said the FDA has granted fast track designation to the oral investigational Bruton's tyrosine kinase inhibitor, rilzabrutinib, which has the potential to be the first BTK inhibitor for the treatment of immune thrombocytopenia. Following positive Phase 1/2 study results, the company said it has initiated a Phase 3 study of the investigational asset.

Separately, the company said the FDA has accepted for priority review the BLA for avalglucosidase alfa for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency). The PDUFA target action is May 18, 2021.

Cellectis Says FDA Lifts Clinical Hold On Early-stage Blood Cancer Study

Cellectis SA CLLS said the FDA has lifted the clinical hold on the Phase 1 MELANI-01 trial evaluating the UCARTCS1 product candidate for the treatment of patients with relapsed or refractory multiple myeloma.

The company said it worked closely with the FDA over the past months, to address the agency's requests, which include adjustments to the MELANI-01 clinical protocol designed to enhance patient safety.

In premarket trading, the stock gained 5.58% to $20.80.

Pfizer, Vivet Announce FDA Nod For Commencing Phase 1/2 Study of Gene Therapy to Treat Liver Disorder

Pfizer Inc. PFE and Vivet Therapeutics, a privately-held gene therapy biotech, said the FDA has cleared Vivet's investigational new drug application for the GATEWAY study, a Phase 1/2 clinical trial evaluating Vivet's proprietary, investigational gene therapy vector, VTX-801, for the potential treatment of Wilson disease, a rare and potentially life-threatening liver disorder. The trial is expected to commence in early 2021.

Pfizer has a minority stake in Vivet and an exclusive option to acquire all outstanding shares. In September, Vivet and Pfizer signed an agreement for the manufacture by Pfizer of the VTX-801 vector for the GATEWAY study.

Pfizer, BioNTech Conclude Vaccine Study, to File For EUA Within Days

Pfizer and BioNTech SE – ADR BNTX announced results from the final efficacy analysis in their ongoing Phase 3 study of their mRNA-based COVID-19 vaccine candidate, BNT162b2, showing it met all of the study's primary efficacy endpoints.

Analysis of the data indicates a vaccine efficacy rate of 95% in participants without prior SARS-CoV-2 infection and also in participants with and without prior SARS-CoV-2 infection, in each case measured from 7 days after the second dose.

The first primary objective analysis is based on 170 cases of COVID-19, as specified in the study protocol, of which 162 cases of COVID-19 were observed in the placebo group versus 8 cases in the BNT162b2 group. Efficacy was consistent across age, gender, race and ethnicity demographics. The observed efficacy in adults over 65 years of age was over 94%.

"To date, the Data Monitoring Committee for the study has not reported any serious safety concerns related to the vaccine," the companies said.

The companies said they plan to file for emergency use authorization within days.

BioNTech shares were rallying 6.74% to $92.79 in premarket trading.

Chiasma Reports Positive Phase 3 Results From Global Study of Acromegaly Drug

Chiasma Inc CHMA announced positive top-line data from its global Phase 3 MPOWERED non-inferiority clinical trial comparing Mycapssa to long-acting injectable somatostatin analogs for maintenance of biochemical response in patients with acromegaly. The MPOWERED trial was designed to support a planned marketing authorization application for Mycapssa in the European Union. Mycapssa has already been approved in the U.S.

In premarket trading, Chiasma shares were gaining 9.41% to $4.42.

Offering

Oncternal Therapeutics Inc ONCT upsized its previously announced offering and purchase on a firm commitment basis of 7.258 million shares of its common stock at $3.10 per share, for raising gross proceeds of $22.5 million.

On The Radar

Earnings

Edap Tms SA EDAP (after the close)