On August 2, 2021 TG Therapeutics, Inc. (NASDAQ: TGTX) reported its financial results for the second quarter ending June 30, 2021 and recent company developments, along with a business outlook for the remainder 2021 (Press release, TG Therapeutics, AUG 2, 2021, View Source [SID1234585521]).

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Michael S. Weiss, the Company’s Chairman and Chief Executive Officer, stated, "We are pleased with the progress made throughout the second quarter, including our ongoing launch of UKONIQ in relapsed or refractory MZL and FL, FDA’s acceptance of our BLA/sNDA for the combination of ublituximab and UKONIQ (U2) to treat CLL and SLL, and the continued advancement of our clinical programs. We have built a strong commercialization infrastructure to launch UKONIQ and have received positive feedback from healthcare providers on their experiences with the product and with the TG team. We believe this solid commercialization foundation will support, if approved, the launch of U2 in CLL and ublituximab in relapsing forms of multiple sclerosis."

Mr. Weiss continued, "We look forward to an exciting second half of 2021, during which we plan to submit a BLA for ublituximab to treat patients with relapsing forms of multiple sclerosis, continue executing on the launch of UKONIQ in MZL and FL, and continue preparations for potential commercialization of U2 in CLL and ublituximab in RMS."

2021 Highlights & Recent Developments

UKONIQ (umbralisib) in Relapsed or Refractory Marginal Zone Lymphoma & Follicular Lymphoma

Launched UKONIQ in the U.S. for the treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one prior anti-CD20 based regimen and adult patients with relapsed or refractory follicular lymphoma (FL) who have received at least three prior lines of systemic therapy.
Generated $2.3M in total net UKONIQ revenue from launch through the end of Q2 2021, approximately 4 months.
Achieved broad U.S. payor coverage for more than 90% of Medicare and commercial lives and inclusion in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines for MZL and FL.
Ublituximab plus UKONIQ (U2) in Chronic Lymphocytic Leukemia

Received FDA acceptance of a Biologics License Application (BLA) for ublituximab and a supplemental New Drug Application (sNDA) for UKONIQ, both submissions requesting approval of U2 as a treatment for patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). These applications were primarily based on results from the UNITY-CLL Phase 3 trial, which included both treatment-naïve and relapsed or refractory (R/R) patients. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 25, 2022 for both applications.
Completed enrollment in the ULTRA-V Phase 2 trial and launched the ULTRA-V Phase 3 randomized trial evaluating the triple combination of U2 plus venetoclax in patients with treatment-naïve and R/R CLL.
Ublituximab in Multiple Sclerosis

Presented positive results from the ULTIMATE I and II Phase 3 trials at the 2021 American Academy of Neurology (AAN) annual meeting and the 7th Congress of the European Academy of Neurology (EAN). Both trials met their primary endpoint with ublituximab treatment demonstrating a statistically significant reduction in annualized relapse rate (ARR) over a 96-week period compared to teriflunomide in patients with relapsing forms of multiple sclerosis (RMS).
TG-1701 in B-cell Malignancies

Presented updated data from TG-1701, our BTK inhibitor, as a monotherapy and in combination with U2 in patients with B-cell malignancies at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, the 2021 European Hematology Association (EHA) (Free EHA Whitepaper) virtual congress and the 16th International Congress on Malignant Lymphoma (ICML).
Remaining Key Objectives for 2021 and Early 2022

Focus on the commercialization of UKONIQ in R/R MZL and FL and expand commercialization capabilities in preparation for a potential launch of U2 in CLL and ublituximab in RMS
Submit a BLA for ublituximab for the treatment of patients with RMS in Q3 2021, based on positive results from the ULTIMATE I and II Phase 3 trials
Obtain approval for U2 in CLL and SLL by the PDUFA goal date of March 25, 2022
Enroll into the newly launched ULTRA-V Phase 3 trial evaluating the triple combination of U2 plus venetoclax
Continue to advance our early pipeline candidates including TG-1501 (cosibelimab), TG-1701 and TG-1801
Financial Results for the Three and Six Months Ended June 30, 2021

Product Revenue, net: Product revenue, net was approximately $1.5 million and $2.3 million for the three and six months ended June 30, 2021, compared to zero during the comparable periods in 2020. Net product revenues represent U.S. sales from our sole commercial product, UKONIQ, which received accelerated approval from the FDA on February 5, 2021.

R&D Expenses: Total research and development (R&D) expense was $44.9 million and $108.0 million for the three and six months ended June 30, 2021, compared to $36.5 million and $72.5 million for the three and six months ended June 30, 2020. The increase was due primarily to licensing milestone fees of approximately $4.0 million and $18.0 million incurred during the three and six months ended June 30, 2021, and an increase in non-cash compensation R&D expense over the comparable periods in 2020.

SG&A Expenses: Total selling, general and administrative (SG&A) expense was $34.0 million and $60.8 million for the three and six months ended June 30, 2021, and $14.4 million and $28.7 million for the three and six months ended June 30, 2020. The increase was due primarily to increased personnel and other selling, general and administrative costs associated with execution of the launch of UKONIQ and planning for the potential launches of U2 in CLL and ublituximab in RMS. We expect our selling, general and administrative expenses to increase for the remainder of 2021 as we continue to prepare for those potential 2022 launches.

Net Loss: Net loss was $78.5 million and $169.1 million for the three and six months ended June 30, 2021, compared to $52.9 million and $104.0 million for the three and six months ended June 30, 2020. Excluding non-cash compensation, the net loss for the three and six months ended June 30, 2021 was approximately $62.2 million and $136.2 million, compared to a net loss of $45.5 million and $85.6 million for the three and six months ended June 30, 2020.

Cash Position and Financial Guidance: Cash, cash equivalents and investment securities were $456.2 million as of June 30, 2021, which the Company believes will be sufficient to fund the Company’s planned operations into 2023.
CONFERENCE CALL INFORMATION
The Company will host a conference call today, August 2, 2021, at 8:30 AM ET, to discuss the Company’s second quarter ended June 30, 2021 financial results and provide a business outlook for the remainder of 2021.

To participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source An audio recording of the conference call will also be available for a period of 30 days after the call.

On August 2, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that it will release its second quarter 2021 financial results on Monday, August 9, after the close of the U.S. financial markets (Press release, Syndax, AUG 2, 2021, View Source [SID1234585520]).

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In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET on Monday, August 9, to discuss the Company’s financial results and provide a general business update.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website, www.syndax.com.

On August 2, 2021 T-knife Therapeutics, Inc., a next-generation T-cell receptor company developing a pipeline of innovative therapeutics for solid tumor patients, reported the successful completion of a $110 million Series B financing (Press release, T-Knife, AUG 2, 2021, View Source [SID1234585519]). The financing was led by Fidelity Management & Research Company, LLC., with participation from other new investors including, LSP, Qatar Investment Authority (QIA), Casdin Capital, Sixty Degree Capital, and CaaS Capital, along with existing investors RA Capital Management, Versant Ventures and founding investor Andera Partners. The company plans to use proceeds from the financing to expand its scientific team, increase manufacturing capacity and advance its pipeline of T-cell receptor (TCR) engineered T cell therapies (TCR-T).

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"Over the past year we have made substantial progress toward our goal of building a leading TCR-T company focused on delivering clinically meaningful benefits for patients with solid tumors," stated Thomas M. Soloway, Chief Executive Officer of T-knife. "We are excited to have the support of this group of dedicated life sciences investors to help us fulfill our mission, and we welcome Dr. Karin Kleinhans of LSP to our board of directors."

"T-knife has an elegant and differentiated approach to identifying potent, cancer-specific TCRs with naturally optimized affinity and specificity profiles, creating a next-generation platform for this promising therapeutic field," said Alex Mayweg, Chairman of T-knife and Managing Director at Versant Ventures. "We are pleased to be progressing TK-8001 toward the clinic and to advance our broader portfolio of product candidates."

T-knife is leveraging its proprietary HuTCR transgenic mouse platform to discover and develop a portfolio of TCR-T programs to treat patients with solid tumors. T-knife’s lead program, TK-8001, is a novel TCR-T product candidate targeting MAGE-A1 positive cancers. T-knife plans to begin enrolling patients in the TK-8001 IMAG1NE Phase 1/2 clinical study in the fourth quarter of 2021 and is planning to submit INDs/CTAs for additional product candidates in 2022.

"The field of TCR-T holds significant promise to change the treatment paradigm for many cancer patients," said Karin Kleinhans, PhD, Partner at LSP who joined T-Knife’s board in connection with the Series B financing. "We are highly encouraged by the progress being made at T-knife to advance its important next-generation therapies."

Olivier Litzka, Partner at Andera Partners, commented, "As a founding investor, it is gratifying to witness the continued success at T-knife. The completion of the Series B financing is an important milestone that will enable us to execute on our vision of building a leading transatlantic immuno-oncology company."

About the HuTCR platform
T cells play a key role in the immune response by directly recognizing and eliminating infected, foreign or altered cells, such as cancer cells. To do this, they use their T-cell receptors (TCRs) to scan the surface of other cells for foreign antigens presented on Human Leukocyte Antigen (HLA) complexes. Cancer cells can be recognized by mutated or viral antigens expressed only in the tumor, or self-antigens normally expressed during embryonic development and in non-somatic adult tissues. Genetic engineering of T cells with TCRs recognizing antigens aberrantly or over-expressed in cancers can redirect these T cells to the tumor, potentially offering curative responses to cancer patients.

The ability to identify potent cancer-specific TCRs has been limiting for the field of TCR-T. In the case of self-antigens, T cells bearing those TCRs are eliminated during T cell development to avoid recognition and attack of healthy tissues. For non-self tumor antigens, such as those derived from viral sequences or mutations, the very low T cell frequency in the blood has limited TCR discovery efforts.

To overcome these challenges, T-knife has developed transgenic mice (HuTCR mice) carrying the human TCRαβ gene loci and expressing multiple human HLAs. Immunizing HuTCR mice with human tumor antigens, for which mice are not tolerant, allows for the identification of both CD4+ and CD8+ T cells with TCRs that have optimized affinity / specificity profiles capable of mediating significant anti-tumor activity. The TCRs from HuTCR mice are of higher affinity for tumor self-antigens than TCRs isolated from human donors and are naturally optimized to maintain a high specificity profile, making HuTCR mice a powerful high-throughput platform for rapidly generating TCRs with best-in-class potential.

On August 2, 2021 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported financial results for the second quarter ending June 30, 2021 and provided a corporate update (Press release, VBI Vaccines, AUG 2, 2021, View Source [SID1234585517]).

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Jeff Baxter, VBI’s President and CEO commented: "The second quarter of 2021 was a data-rich period for the Company, with positive results across our lead programs, leading to meaningful advancement of our pipeline. As a result of the encouraging data reported in Q2, the FDA granted Fast Track Designation for VBI-1901 in recurrent GBM, we demonstrated the potency of the eVLP particulate delivery platform against COVID-19, supporting the assessment of our COVID-19 Beta variant vaccine candidate in the next phase of development, and we initiated a first-in-class Phase 2 combination study assessing a potential functional cure regimen for chronic HBV infection. Additionally, as we advance towards our PDUFA target action date for our 3-antigen prophylactic HBV vaccine candidate, we continue to lay the groundwork for the commercial launch, subject to regulatory approvals. The AMA established a unique CPT Code for a 3-antigen (S, Pre-S1, Pre-S2) Hepatitis B (HBV) Vaccine, a meaningful milestone as it reinforces the scientific differentiation of our candidate and enables a streamlined reimbursement process. At the beginning of the year, we laid out an ambitious and aggressive plan for the development of our pipeline in 2021, and in the first half of the year we’ve successfully executed against that plan. We look forward to sharing upcoming data and regulatory milestones as we move into the second half of 2021."

Q2 2021 Key Program Achievements and Anticipated Upcoming Milestones

Hepatitis B (HBV)
3-Antigen HBV Prophylactic Vaccine Candidate

November 30, 2021: U.S. Prescription Drug User Fee Act (PDUFA) target action date set by U.S. Food and Drug Administration (FDA)
European Medicines Agency (EMA) regulatory review ongoing, following acceptance of Marketing Authorization Application (MAA) filing in December 2020
July 2021: The American Medical Association (AMA) Current Procedural Terminology (CPT) Panel established a unique CPT code for a 3-antigen (S, Pre-S1, Pre-S2) Hepatitis B (HBV) vaccine, under which VBI’s vaccine candidate will be reported if approved
Submissions to United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) and to Health Canada are in process and are expected to be completed in 2021
VBI-2601 (BRII-179) : HBV Immunotherapeutic Candidate

April and June 2021: Complete Phase 1b/2a study dataset announced – EASL presentation selected for inclusion in the "Best of the International Liver Congress" summary, highlighting the most noteworthy contributions to the scientific program. Data demonstrated:
VBI-2601 (BRII-179) induced both B cell (antibody) and T cell responses in chronically-infected HBV patients and was well-tolerated with no safety signals observed
Evidence to support further clinical evaluation of VBI-2601 (BRII-179) as an immunomodulator in combination with other treatment modalities as a potential functional cure for chronic HBV infection
April 2021: Phase 2 combination study of VBI-2601 (BRII-179) and BRII-835 (VIR-2218), Vir Biotechnology’s investigational HBV-targeting siRNA, initiated by partner, Brii Biosciences (Brii Bio)
COVID-19 & Coronaviruses
VBI-2900 : eVLP Coronavirus Vaccine Program

June 2021: Initial positive Phase 1 data of VBI-2902 announced, demonstrating that a 5µg dose, which expresses an optimized form of the SARS-CoV-2 spike antigen and is adjuvanted with aluminum phosphate, was generally well-tolerated and elicited a potent immune response significantly higher than that seen in human convalescent sera. Notably:
Safety and tolerability: VBI-2902 was well-tolerated with no safety signals observed, and no increase in reactogenicity with subsequent doses
Neutralizing titers: After two doses, VBI-2902 induced neutralization titers in 100% of participants, with 4.3x the geometric mean titer (GMT) compared to convalescent sera
Antibody binding titers: After two doses, VBI-2902 induced antibody binding titers in 100% of participants, with peak antibody binding GMT of 1:4,047 – 5.0x the GMT compared to convalescent sera
Q3 2021 : As a result of this platform-validating initial data, and in response to the increased circulation of COVID-19 variants, the next phase of the ongoing adaptive Phase 1/2 study, expected to initiate in Q3 2021, will assess VBI-2905, VBI’s eVLP vaccine candidate directed against the COVID-19 Beta variant
H1 2022 : Expected initiation of the first clinical study of VBI’s multivalent vaccine candidate, designed to increase the breadth of protection against known and emerging variants of COVID-19
Glioblastoma (GBM)
VBI-1901: Cancer Vaccine Immunotherapeutic Candidate

June 2021: Positive data from Phase 2a (Part B) of ongoing Phase 1/2a study presented at ASCO (Free ASCO Whitepaper), including:
VBI-1901 + GM-CSF study arm:
6-month and 12-month OS: 80% (n=8/10) and 60% (n=6/10), compared to historical controls of 60% and 30%1, respectively
2 durable partial responses (≥ 50% tumor reduction) and 2 stable disease observations – 40% disease control rate
VBI-1901 + GSK’s AS012 adjuvant system study arm:
6-month OS: 89% (n=8/9); 12-month OS timepoint not yet reached
5 stable disease observations – 50% disease control rate
June 2021: FDA Fast Track Designation granted for VBI-1901 + GM-CSF in first recurrent GBM patients
Q4 2021: Expected initiation of a randomized, controlled clinical study with registration potential
Recent Peer-Reviewed Publications

May 2021: The Lancet Infectious Diseases publication of data from PROTECT – Phase 3 immunogenicity and safety study assessing VBI’s 3-antigen HBV vaccine candidate compared to Engerix-B in adults age 18 and older. Link to publication can be found here.
June 2021: Vaccine publication of data from a Phase 3 immunogenicity and safety study, conducted in Vietnam and completed in 2007, assessing VBI’s 3-antigen HBV vaccine candidate in healthy Asian adults. Link to publication can be found here.
July 2021: Vaccine publication of data from VBI-2902a preclinical and challenge COVID-19 studies. Link to publication can be found here.
Additional Corporate Updates

Board Appointment: In July 2021, VBI appointed Linda Bain, CFO of Codiak BioSciences, Inc., to the Company’s Board of Directors
Debt Financing: In May 2021, VBI drew down a $12 million second tranche under its debt financing facility with K2 Healthventures, bringing the amount drawn to-date to $32 million
Second Quarter 2021 Financial Results

Cash Position: VBI ended the second quarter of 2021 with $135.0 million in cash, cash equivalents, and short-term investments compared with $119.1 million as of December 31, 2020.
Net Cash Used in Operating Activities: Net cash used in operating activities for the six months ended June 30, 2021 was $17.4 million, compared to $15.5 million for the same period in 2020. The increase in cash outflows is largely a result of an increase in net loss, offset by the change in operating working capital, notably the cash received in advance from the CEPI funding agreement.
Cash Used for Purchase of Property and Equipment: Cash used for the purchase of property and equipment was $1.0 million for the six months ended June 30, 2021, compared to $0.3 million for the same period in 2020. The increase is a result of routine purchases of property and equipment in Rehovot, Israel.
Revenue: Revenue in the second quarter of 2021 was $0.1 million, compared to $0.2 million for the same period in 2020. The decrease in revenues was due to a decrease in R&D services revenue as part of the collaboration with Brii Bio. Fewer manufacturing and non-clinical research services were required in the three months ending June 30, 2021 compared to the three months ending June 30, 2020.
Cost of Revenues: Cost of revenues was $2.6 million in the second quarter of 2021 as compared to $2.1 million for the same period in 2020. The increase in the cost of revenues was due to increased outsourced testing costs and inventory-related costs incurred in the three months ending June 30, 2021 compared to the same period in 2020.
Research and Development (R&D): R&D expenses for the second quarter of 2021 were $4.6 million compared to $2.4 million in the second quarter of 2020. The increase was a result of increased expenses related to our coronavirus vaccine program, including the Phase 1 clinical study of VBI-2902 and development and manufacture of VBI-2905, offset by government grants and funding arrangements. The increase was also due to an increase in R&D expenses related to the development of our other vaccine candidates, which was offset by a decrease in regulatory costs related to the BLA submission for the 3-antigen prophylactic HBV vaccine candidate that was submitted in 2020.
General and Administrative (G&A): G&A expenses were $9.4 million for the second quarter of 2021, compared to $3.9 million for the same period in 2020. The increase was a result of the increased pre-commercialization activities related to the 3-antigen prophylactic HBV vaccine candidate in preparation for potential regulatory approvals, in addition to increased insurance and labor costs.
Net Loss: Net Loss and net loss per share for the second quarter of 2021 were $17.5 million and $0.07, respectively, compared to a net loss of $9.5 million and a net loss per share of $0.04 for the second quarter of 2020.

On August 2, 2021 Immunovant, Inc. (Nasdaq: IMVT), a clinical-stage biopharmaceutical company focused on enabling normal lives for people with autoimmune diseases, reported that it has received a $200 million strategic investment from Roivant Sciences (Press release, Roivant Sciences, AUG 2, 2021, https://investors.immunovant.com/news-releases/news-release-details/immunovant-receives-200-million-strategic-investment-roivant [SID1234585515]). Immunovant intends to use the proceeds from this investment to advance the development of IMVT-1401 in multiple indications.

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Roivant has purchased 17,021,276 shares of Immunovant’s common stock at a price of $11.75 per share, which purchase has been approved by a special committee of Immunovant directors not affiliated with Roivant. This represents approximately a 15% premium to Immunovant’s 20 trading day volume weighted average price. After giving effect to the investment, Immunovant has a pro forma cash balance of approximately $600 million and Roivant has increased its ownership stake in Immunovant from 57.5% to 63.8%, based on Immunovant’s cash balance and share count as of March 31, 2021.

"We are excited to announce this significant investment by Roivant, which will expedite our development of IMVT-1401 for a wide range of autoimmune disorders," said Dr. Pete Salzmann, Chief Executive Officer of Immunovant. "Over the next 12 months, we plan to initiate a pivotal trial for myasthenia gravis, resume our trials in WAIHA and TED and initiate at least two additional clinical studies, including another pivotal trial in 2022."

"Roivant and Immunovant explored a range of possible transactions over the past few months, including a potential acquisition by Roivant of the minority interest in Immunovant, and ultimately agreed on this significant investment in order to support a robust development plan for IMVT-1401 and increase our stake in the company," said Matt Gline, Chief Executive Officer of Roivant Sciences. "We are incredibly excited about the prospects for IMVT-1401, and we are eager to support Immunovant through this investment. We look forward to continuing to work closely with Dr. Salzmann and the Immunovant management team to help develop IMVT-1401 to maximize benefit for patients with high levels of unmet medical need."