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

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

Biotech stocks headed higher in the week ended Dec. 5 as broader market optimism pervaded into the sector. The sector also continued to get support from vaccine-related positive news flow.

The biggest headline of the week was the first-ever emergency-use authorization of a coronavirus vaccine candidate. The U.K. became the first country to conditionally approve a vaccine, as the nation's regulator gave its nod to Pfizer Inc. PFE and BioNTech SE – ADR BNTX for their mRNA vaccine, BNT162b2. The timeline for authorizations elsewhere has also been set.

Moderna Inc MRNA reported at the start of the week positive primary efficacy and safety data, and also filed for emergency use authorization with the U.S. Food and Drug Administration.

Meanwhile, Novavax, Inc. NVAX hinted at a delay in the U.S. leg of the Phase 3 study of its vaccine candidate. And Inovio Pharmaceuticals Inc INO, which also is working on a vaccine, added a new contract development and manufacturing organization (CDMO) to its consortium of partners.

The two FDA decisions slated for the week led to positive results, with the agency approving Vanda Pharmaceuticals Inc.'s VNDA sleep disorder drug and BioCryst Pharmaceuticals, Inc.'s BCRX treatment for hereditary angioedema.

The week also witnessed multiple presentations at the virtual American Society of Hematology annual meeting.

Four biotechs made their Wall Street debuts this week, raising a combined $782.5 billion in gross proceeds from initial public offerings.

Here are the key catalysts for the upcoming week:

FDA Meeting On Pfizer-BioNTech Vaccine

The FDA's Vaccines and Related Biological Products Advisory Committee will meet Dec. 10 (Thursday) in an open session to discuss emergency use authorization of the vaccine developed by Pfizer and BioNTech. This is one of the most highly anticipated points in the timeline of this unfolding COVID-19 vaccine saga. It could determine whether some Americans start getting vaccines as soon as the next day. Vaccinations are set to begin in the U.K. on Tuesday.


American Epilepsy Society, or AES, Annual Meeting (virtual event): Dec. 4-8
62nd American Society of Hematology, or ASH, Annual Meeting and Exposition virtual event: Dec. 5-8
San Antonio Breast Cancer Symposium, or SABCS, being held virtually: December 8-11
ESMO Immuno-oncology Virtual Congress: Dec. 9-12

Clinical Readouts/Presentations

Sarepta Therapeutics Inc SRPT will present on Monday interim data from the MOMENTUM study, a multiple-ascending dose clinical trial of SRP-5051, for the treatment of Duchenne muscular dystrophy.

ASH Conference Presentations


Erytech Pharma SA ERYP: results of Phase 2 trial of eryaspase in acute lymphoblastic leukemia patients

Imara Inc IMRA: data from the ongoing IMR-687 Phase 2a open label extension clinical trial in adult patients with sickle cell disease

Blueprint Medicines Corp BPMC: Overall survival data in patients with advanced systemic mastocytosis receiving avapritinib in the Phase I EXPLORER study

Protagonist Therapeutics Inc PTGX: updated results from the Phase 2 proof-of-concept study of PTG-300 in polycythemia vera

Xencor Inc XNCR: Updated results from Phase 1 studies of vibecotamab in acute myeloid leukemia

Rocket Pharmaceuticals Inc RCKT: Phase 1 study of lentiviral mediated gene therapy for pyruvate kinase deficiency

Beigene Ltd BGNE: initial results from the MAGNOLIA Phase 2 trial of zanubrutinib in patients with relapsed/refractory marginal zone lymphoma

MacroGenics Inc MGNX: Phase 1/2 data for flotetuzumab in primary induction failure and early relapse acute myeloid leukemia

Vertex Pharmaceuticals Incorporated VRTX & Crispr Therapeutics AG CRSP: results from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with sickle cell disease and beta thalassemia.

ADC Therapeutics SA ADCT: preliminary results of a Phase 2 study of camidanlumab tesirine (Cami), an antibody-drug conjugate, in patients with relapsed or refractory Hodgkin lymphoma; interim results of loncastuximab tesirine combined with ibrutinib in relapsed/refractory diffuse large B-cell lymphoma

Syndax Pharmaceuticals Inc SNDX: updated data from the Phase 1 trial of axatilimab, its anti-CSF-1R monoclonal antibody, in patients with chronic graft versus host disease

CTI BioPharma Corp CTIC: results from the Phase 1/2 study of pacritinib in graft-versus-host disease

Aptose Biosciences Inc APTO: data from the Phase 1a/b dose escalation study of CG-806 in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma or Non-Hodgkin's Lymphomas

Johnson & Johnson JNJ & Halozyme Therapeutics, Inc. HALO: primary data from the Phase 3 APOLLO study of subcutaneous daratumumab in combination with pomalidomide and dexamethasone in patients with multiple myeloma who have received one or more prior lines of therapy

Humanigen Inc HGEN: results from the Phase 1/2 multi-center study of lenzilumab and axicabtagene ciloleucel in patients with relapsed or refractory large B cell lymphoma

Imv Inc IMV: updated data from phase 2 SPiReL study evaluating IMV's T cell therapy - DPX-Survivac - in combination with Merck & Co., Inc.'s MRK Keytruda in patients with relapsed / refractory diffuse large B cell lymphoma

Related Link: What Pfizer, BioNTech Investors Need To Know About COVID-19 Vaccine With Conditional UK Approval


Bluebird bio Inc BLUE: updated results from patients in Group C of the Phase 1/2 HGB-206 study of lentiGlobin for sickle cell disease gene therapy

Rocket Pharmaceuticals: Phase 1/2 study of lentiviral-mediated ex-vivo gene therapy for pediatric patients with severe leukocyte adhesion deficiency-I and updated results from global clinical studies of RP-L102 in Fanconi anemia

Beigene: data from the Phase 2 study of zanubrutinib in patients with Waldenström macroglobulinemia as well as Phase 2 study of zanubrutinib in patients with relapsed/refractory B-cell malignancies

Sutro Biopharma Inc STRO: additional Phase 1 dose-escalation data for STRO-001 in patients with B-cell non-Hodgkin lymphoma

Precigen Inc PGEN: initial Phase 1 data for PRGN-3006 in myelodysplastic syndromes and acute myeloid leukemia

Forma Therapeutics Holdings Inc FMTX: data from the Phase 1 study of FT-4202 in patients with Sickle Cell Disease

Autolus Therapeutics PLC AUTL: longer term follow-up data for AUTO1 in acute lymphoblastic leukemia, and updated Phase 2 data and longer-term follow up data for AUTO3 in diffuse large B cell lymphoma

Curis, Inc. CRIS: results (pharmacokinetics and activity) from the Phase 1 study of CA-4948 in patient with relapsed or refractory hematologic malignancies, and results from the Phase 1 open-label dose escalation trial evaluating CA-4948 in patients with acute myelogenous leukemia or myelodysplastic syndrome

TG Therapeutics Inc common stock TGTX: results from the Phase 3 UNITY-CLL study of umbralisib plus ublituximab in patients with treatment naïve and relapsed/refractory chronic lymphocytic leukemia, and results from the Phase 2 global UNITY-NHL trial that is evaluating umbralisib in patients with relapsed or refractory indolent non-Hodgkin's lymphoma

Agios Pharmaceuticals Inc AGIO: Phase 1 multiple ascending dose study data for mitapivat in subjects with sickle cell disease

Orchard Therapeutics PLC – ADR ORTX: data from the first patient treated in the proof-of-concept study of OTL-201, an investigational ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of mucopolysaccharidosis type IIIA

Trillium Therapeutics Inc TRIL: updates from Phase 1 dose escalation and expansion study of TTI-621, a biologic targeting CD47, in patients with relapsed or refractory hematologic malignancies

Allovir Inc ALVR: results from a Phase 2 study of ALVR106 for treating severe, drug-refractory viral infections in patients following hematopoietic stem cell transplantation

GlycoMimetics Inc GLYC and Pfizer: Phase 3 data for rivipansel in vaso-occlusive crisis of sickle cell disease

Alexion Pharmaceuticals, Inc. ALXN and Fortress Biotech FBIO: Phase 2 dose selection study data for CAEL-101 in patients with AL amyloidosis

Oncternal Therapeutics Inc ONCT: data update for patients with mantle cell lymphoma and chronic lymphocytic leukemia, treated with cirmtuzumab plus ibrutinib in a Phase 1/2 study


Uniqure NV QURE: first data from the Phase 3 HOPE-B gene therapy trial evaluating efficacy and safety 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 (11:45 am ET)

SABCS Presentations

Infinity Pharmaceuticals Inc. INFI and Arcus Biosciences Inc RCUS: Efficacy and safety of Arcus' etrumadenant + pegylated liposomal doxorubicin ± Infinity's eganelisib in participants with metastatic ovarian and triple negative breast cancer (Wednesday, 2 a.m. ET)

Infinity Pharma: Poster presentation on initial Phase 2 data from the study evaluating a triple combo of Infinity's eganelisib, Roche Holdings AG's Basel ADR Common Stock RHHBY Tecentriq and Bristol-Myers Squibb Co BMY chemotherapy medication Arabaxane as first-line therapy for locally advanced or metastatic triple-negative breast cancer (Wednesday, 2 a.m. ET)

Evelo Biosciences Inc EVLO: Poster presentation of additional data from Phase 1/2 trial of EDP1503 in triple-negative breast cancer

Athenex Inc ATNX: Phase 2 data for oral Paclitaxel and Encequidar in the treatment of cutaneous angiosarcoma, the breast angiosarcoma group

Incyte Corporation INCY & Oncolytics Biotech, Inc. ONCY: poster presentation of phase 2 data for Incyte's retifanlimab and the oncolytic virus pelareorep in metastatic triple negative breast cancer (Wednesday, 9 a.m. ET)

Oncolytics Biotech: Phase 2 study to assess overall response rate by inducing an inflammatory phenotype in metastatic breast cancer with the oncolytic reovirus pelareorep in combination with anti-PD-L1 avelumab and paclitaxel

G1 Therapeutics Inc GTHX: final analysis of Phase 2 data for trilaciclib in combination of gemcitabine/carboplatin in patients with metastatic triple-negative breast cancer (Wednesday, 10 a.m. to 11:15 a.m.)

Puma Biotechnology Inc PBYI: Latest findings from the breast cancer cohort in the SUMMIT Phase 2 study of 'basket' trial of neratinib + trastuzumab + fulvestrant for HER2-mutant, HR-positive, metastatic breast cancer; Final overall survival analysis from the phase 3 study of neratinib in patients with HER2-positive early-stage breast cancer (both on Wednesday)

ESMO Presentations

Innate Pharma SA IPHA: updated results from a Phase 2 study investigating the combination of monalizumab and cetuximab in patients with recurrent or metastatic head and neck squamous cell cancer (Wednesday)


Veru Inc VERU (Wednesday, before the market open)
Enzo Biochem, Inc. ENZ (Wednesday, after the close)

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

Posted In: American Society of HematologyCovid-19emergency usePfizerBiotechEarningsNewsPenny StocksSmall CapFDA