Biotech week ahead, Dec. 7

 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 0.65% and BioNTech SE – ADR BNTX 1.11% for their mRNA vaccine, BNT162b2. The timeline for authorizations elsewhere has also been set.

Moderna Inc MRNA 3.24% 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 2.25% hinted at a delay in the U.S. leg of the Phase 3 study of its vaccine candidate. And Inovio Pharmaceuticals Inc INO 0.64%, 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 2.06% sleep disorder drug and BioCryst Pharmaceuticals, Inc.'s BCRX 18.79% 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.

Conferences

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 3.13% 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

Sunday

Erytech Pharma SA ERYP 3.48%: results of Phase 2 trial of eryaspase in acute lymphoblastic leukemia patients

Imara Inc IMRA 1.51%: data from the ongoing IMR-687 Phase 2a open label extension clinical trial in adult patients with sickle cell disease

Blueprint Medicines Corp BPMC 3.1%: Overall survival data in patients with advanced systemic mastocytosis receiving avapritinib in the Phase I EXPLORER study

Protagonist Therapeutics Inc PTGX 5.19%: updated results from the Phase 2 proof-of-concept study of PTG-300 in polycythemia vera

Xencor Inc XNCR 0.82%: Updated results from Phase 1 studies of vibecotamab in acute myeloid leukemia

Rocket Pharmaceuticals Inc RCKT 4.95%: Phase 1 study of lentiviral mediated gene therapy for pyruvate kinase deficiency

Beigene Ltd BGNE 0.71%: initial results from the MAGNOLIA Phase 2 trial of zanubrutinib in patients with relapsed/refractory marginal zone lymphoma

MacroGenics Inc MGNX 6.24%: Phase 1/2 data for flotetuzumab in primary induction failure and early relapse acute myeloid leukemia

Vertex Pharmaceuticals Incorporated VRTX 0.64% & Crispr Therapeutics AG CRSP 5.04%: 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 0.87%: 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 1.78%: 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 3.01%: 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 0.82% & Halozyme Therapeutics, Inc. HALO 2.27%: 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 5.81%: 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 1.14%: updated data from phase 2 SPiReL study evaluating IMV's T cell therapy - DPX-Survivac - in combination with Merck & Co., Inc.'s MRK 0.61% Keytruda in patients with relapsed / refractory diffuse large B cell lymphoma

Monday

Bluebird bio Inc BLUE 0.13%: 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 33.24%: additional Phase 1 dose-escalation data for STRO-001 in patients with B-cell non-Hodgkin lymphoma

Precigen Inc PGEN 0.06%: initial Phase 1 data for PRGN-3006 in myelodysplastic syndromes and acute myeloid leukemia

Forma Therapeutics Holdings Inc FMTX 0.07%: data from the Phase 1 study of FT-4202 in patients with Sickle Cell Disease

Autolus Therapeutics PLC AUTL 0.57%: 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 6%: 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 3.06%: 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 1.22%: Phase 1 multiple ascending dose study data for mitapivat in subjects with sickle cell disease

Orchard Therapeutics PLC – ADR ORTX 2.14%: 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 5.8%: 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 6.22%: 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 2.47% and Pfizer: Phase 3 data for rivipansel in vaso-occlusive crisis of sickle cell disease

Alexion Pharmaceuticals, Inc. ALXN 0.45% and Fortress Biotech FBIO 1.83%: Phase 2 dose selection study data for CAEL-101 in patients with AL amyloidosis

Oncternal Therapeutics Inc ONCT 4.72%: data update for patients with mantle cell lymphoma and chronic lymphocytic leukemia, treated with cirmtuzumab plus ibrutinib in a Phase 1/2 study

Tuesday

Uniqure NV QURE 0.79%: 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 0.69% and Arcus Biosciences Inc RCUS 3.64%: 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 2.2% Tecentriq and Bristol-Myers Squibb Co BMY 0.86% 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 4.18%: Poster presentation of additional data from Phase 1/2 trial of EDP1503 in triple-negative breast cancer

Athenex Inc ATNX 2.28%: Phase 2 data for oral Paclitaxel and Encequidar in the treatment of cutaneous angiosarcoma, the breast angiosarcoma group

Incyte Corporation INCY 0.29% & Oncolytics Biotech, Inc. ONCY 11.02%: 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 4.99%: 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 5.03%: 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 7.16%: 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)

Earnings

Veru Inc VERU 0.61% (Wednesday, before the market open)
Enzo Biochem, Inc. ENZ 10.5% (Wednesday, after the close)

https://www.benzinga.com/general/biotech/20/12/18663084/week-ahead-in-biotech-fda-to-decide-on-emergency-use-of-pfizers-covid-vaccine-hematology-conferen

What's new at San Antonio Breast Cancer Symposium this year

 The "hottest" presentation at the upcoming 2020 San Antonio Breast Cancer Symposium (SABCS) comes from RxPONDER (abstract GS3-00), a major randomized clinical trial assessing use of a recurrence score among women with lymph node-positive, early-stage breast cancer to determine who might safely forgo chemotherapy.

That's the word from Virginia Kaklamani, MD, from the University of Texas Health Sciences Center San Antonio. Kaklamani, a professor of medicine in the Division of Hematology/Oncology, is co-director of the meeting that runs online December 8-11.

If the new trial sounds familiar, that's because it's a lot like the TAILORx trial, the results of which were first presented in 2018 and have changed practice in women with early-stage disease and no lymph node involvement.

"This is the lymph-node positive TAILORx. It's extremely important," Kaklamani told Medscape Medical News, adding that both trials involved women with HR-positive, HER2-negative disease.

If the RxPONDER trial, she explained, shows similar outcomes between women randomized to adjuvant endocrine therapy alone vs endocrine therapy plus chemotherapy, then clinicians "can potentially avoid giving chemotherapy to a large number of women who are currently receiving it."

Because women with nodal involvement (one to three positive axillary nodes) are at a higher risk of recurrence, RxPONDER may provide needed insight on the management of these types of breast cancers, Kaklamani suggested.

Both trials have used the 21-tumor gene expression assay (Oncotype Dx) to determine recurrence-risk status.

Kaklamani also spotlighted the phase 3 CONTESSA trial (abstract GS4-01) in 600+ patients with locally advanced or metastatic breast cancer that is HR-positive and HER2-negative and has been previously treated with a taxane.

The trial features an experimental oral taxane, tesetaxel. The primary objective is to compare the efficacy of tesetaxel plus a reduced dose of capecitabine (Xeloda) vs the approved dose of capecitabine alone. Presented data will include progression-free survival (PFS) results, indicating about a 3-month PFS advantage with tesetaxel, which is taken once every 3 weeks.

"Oral drugs are convenient for patients and, despite limitations, they are, all in all, a revolution in cancer treatment," noted Kaklamani, adding that they beneficially eliminate the need for time-consuming infusions and related clinic visits as well as drug ports. 

It will be interesting to see what Steven Vogl, MD, a private practitioner in the Bronx, New York City, has to say about CONTESSA and the rest of the SABCS.

He is usually a commentator from the meeting floor, whose self-introduction, "Vogl, New York," is well-known to perennial meeting attendees, as reported in a Medscape Medical News profile piece some years ago.

This year the medical oncologist will also serve as the chair of the "View from the Trenches" session, which is devoted to summarizing the meeting's most important findings for everyday practitioners.

A number of years ago, Vogl proposed the idea of this where-the-rubber-meets-the-road session to SABCS meeting planners, which they then adopted. This year, Kaklamani invited Vogl to run the session and he accepted.

Vogl is a "really smart guy who is always right on" with his comments and questions, and he will be the first-ever independent, community-based oncologist to chair a meeting session, said Kaklamani.

Other Hot Topics

Another hot topic featured at the meeting will be the use of CDK4/6 inhibitors in the adjuvant treatment of HR-positive and HER2-negative disease that has a high-risk of recurrence, Kaklamani said. New data from two trials, monarchE and PENELOPE-B, will be presented.

First, there will be an update from the monarchE trial (abstract GS1-01). The first results from this trial were reported in September at the ESMO Virtual Congress 2020 and featured abemaciclib (Verzenio). The positive outcome represented the first treatment improvement in this high-risk setting in more than 20 years, according to experts.

A similar trial, PENELOPE-B (abstract GS1-02), looks at palbociclib (Ibrance) in a somewhat different population — those patients with high relapse risk after neoadjuvant chemotherapy. "These are even higher risk ER+ patients [than those in monarchE], which is why they received chemotherapy before surgery," commented Kaklamani.

In triple negative disease, there will be overall survival (OS) results from the phase 3 KEYNOTE-355 study (abstract GS3-01) of pembrolizumab (Keytruda) vs placebo (plus chemotherapy for all patients) as first-line therapy for locally recurrent inoperable or metastatic triple-negative breast cancer. "It's potentially a huge deal," said Kaklamani about the OS data, if they are statistically significant.

A meta-analysis (abstract GS4-08) of data on circulating tumor cells (CTCs), which are shed from the primary tumor into the bloodstream, may point to their value as a tool to determine whether or not a breast cancer treatment is effective. "CTCs allow you to assess how a treatment is doing before you do scans, which typically occur 3 months or so later," explained Kaklamani.

CTC results can be assessed in 3 to 4 weeks and allow clinicians to change treatments if CTC volume increases. However, a previous study of CTCs did not show a clinical benefit with the tool among patients treated mainly with chemotherapies. What's different about the new study, which is from an international group of investigators, is in the treatments patients with metastatic breast cancer received. "This study is from a different era — with targeted therapies," said Kaklamani.

In the new study, changes in CTC levels (with a reduction being a good result) between baseline (pretreatment) and follow-up were analyzed to determine whether they were associated with overall survival.

COVID Sessions

On the meeting's first day, SABCS will feature a special session on COVID-19 and breast cancer. The meeting organizers sought to separate the wheat from the chaff in this subject, as much has already been written, published, or presented.

"We received a lot of abstracts on COVID that were studies that were poorly done. We tried to tease through them and select the well-researched ones," acknowledged Kaklamani.

The organizers included two patient advocates who have had COVID-19, including during treatment for breast cancer, as participants in the meeting session. The session will also feature global perspectives, with presenters from Brazil, Italy, and the Netherlands. 

Plenary Lectures

The meeting's two plenary lectures will focus, respectively, on the increasingly used clinical approach of neoadjuvant therapy in breast cancer, and research in the time of a pandemic.

Elizabeth Mittendorf, MD, PhD, a surgical oncologist and director of the Breast lmmuno-Oncology program and co-director of the Breast Cancer Clinical Research Program at the Dana-Farber/Brigham and Women's Cancer Center, Boston, will present "Local Regional Management Following Neoadjuvant Therapy: Minding the Knowledge Gaps."

Ned Sharpless, MD, director of the National Cancer Institute (NCI), will present "Advancing Cancer Research During Challenging Times."

Kaklamani has disclosed consulting fees with Amgen, Eisai, Puma, Celldex, AstraZeneca, and Athenex; fees for non-CME services received directly from commercial interest or their agents from Pfizer, Celgene, Genentech, Genomic Health, Puma, Eisai, and Novartis; and contracted research with Eisai.

https://www.medscape.com/viewarticle/942048

Pfizer said to seek emergency approval for COVID-19 vaccine in India

 Pfizer Inc has applied for emergency use authorisation of its coronavirus vaccine in India, media said on Sunday, the first to do so in a country with the world’s second-highest number of infections.

The U.S. company, whose vaccine was recently approved by the British government, has written to the Drugs Controller General of India (DCGI), CNN-News18 reported citing a government source.

Officials at the DCGI and the health ministry did not respond to requests for comment. A government official, however, told Reuters that no application had been received as of Saturday night.

Indian officials have said they are pinning their hopes mainly on locally tested vaccines instead of those developed by Pfizer and Moderna Inc.

The Pfizer shot needs to be stored at minus 70 degrees Celsius (-94 F) or below, temperatures that most Indian cold storages cannot reach.

India has reported more 9.57 million COVID-19 cases, behind only the United States, with nearly 140,000 deaths.

https://www.reuters.com/article/us-health-coronavirus-pfizer-india/pfizer-seeks-emergency-approval-for-covid-19-vaccine-in-india-media-say-idUSKBN28G037