On March 1, 2022 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the fourth quarter ended December 31, 2021. In addition, the Company provided a clinical and business update (Press release, Syndax, MAR 1, 2022, View Source [SID1234609275]).

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"2021 was marked by substantial progress advancing our pipeline of novel cancer therapies, including the initiation of our two pivotal programs for which we expect to report topline data starting in the first half of 2023," said Michael A. Metzger, Chief Executive Officer. "We believe SNDX-5613 is poised to serve as a first-to-market and best-in-class menin inhibitor for patients with genetically defined acute leukemias. With our first regulatory filing for SNDX-5613 expected in 2023, we are keenly focused on laying the groundwork for the potential commercial launch, while concurrently expanding into the frontline and maintenance settings, with three new trials expected to begin in the first half of this year."

Mr. Metzger continued, "Enrollment remains on track in our global pivotal Phase 2 AGAVE-201 trial of axatilimab in chronic graft-versus-host disease (cGVHD), with topline data expected in the first half of 2023 and a potential Biologic License Application (BLA) filing in 2023. Beyond cGVHD, we are also working to unlock axatilimab’s full potential in additional fibrotic diseases where the monocyte-macrophage lineage plays a vital role, with commencement of a Phase 2 trial in idiopathic pulmonary fibrosis (IPF) expected in the fourth quarter of this year."

Recent Pipeline Progress and Anticipated Milestones

SNDX-5613

During an oral presentation at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2021, the Company reported positive data demonstrating continued robust clinical activity with durable responses in the Phase 1 portion of the AUGMENT-101 trial of SNDX-5613, the Company’s highly selective oral menin inhibitor, in relapsed/refractory (R/R) patients with mutant nucleophosmin (NPM1) or mixed lineage leukemia rearranged (MLLr) acute leukemias.
The pivotal Phase 2 portion of AUGMENT-101 is ongoing and the Company expects to complete enrollment in at least one of the three pivotal cohorts later this year. The trials are expected to enroll a total of 64 adult and up to 10 pediatric patients across each of three distinct trial populations: patients with NPM1 mutant acute myeloid leukemia (AML), patients with MLLr AML, and patients with MLLr acute lymphocytic leukemia (ALL). Based on discussions with the U.S. Food and Drug Administration (FDA), AUGMENT-101 may serve as the basis for regulatory filings in each of the three distinct populations. The Company expects to receive initial topline data from the trials starting in the first half of 2023, with the potential for the first NDA filing in 2023.
In December 2021, the Company announced that the European Commission granted Orphan Drug Designation (ODD) to SNDX-5613 for the treatment of AML. SNDX-5613 was previously granted ODD for the treatment of adult and pediatric AML by the U.S. FDA.
The Company expects to initiate three additional trials in the first half of 2022 to assess the safety, tolerability, and preliminary anti-leukemic efficacy of SNDX-5613 in combination with venetoclax and azacitidine as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial, in combination with chemotherapy in patients with R/R NPM1 or MLLr acute leukemias in the AUGMENT-102 trial, and in NPM1 or MLLr patients with measurable residual disease progression following initial treatment as part of the Australian Leukemia and Lymphoma Group (ALLG) INTERCEPT Master Clinical Trial.
Axatilimab

In December 2021, the Company reported updated positive data demonstrating broad activity and tolerability of axatilimab, its anti-CSF-1R monoclonal antibody, in a Phase 1/2 trial in patients with cGVHD. The data were presented during an oral session at the 63rd ASH (Free ASH Whitepaper) Annual Meeting.
Enrollment is ongoing in the Company’s global pivotal Phase 2 AGAVE-201 trial of axatilimab in patients with cGVHD, with topline data expected in the first half of 2023. The trial is evaluating the safety and efficacy of three doses and schedules of axatilimab. The primary endpoint will assess objective response rate based on the 2014 NIH consensus criteria for cGVHD, with key secondary endpoints including duration of response and improvement in modified Lee Symptom Scale score. Topline data from the trial are expected in the first half of 2023, with the potential for a BLA filing in 2023.
In December 2021, Syndax and Incyte closed its previously announced exclusive worldwide collaboration and license agreement to develop and commercialize axatilimab. Closing of the agreement triggered a $117 million upfront payment by Incyte to Syndax, as well as Incyte’s $35 million equity investment in Syndax.
Corporate Updates

Earlier today, the Company announced the appointment of Kate Madigan, M.D., as Chief Medical Officer. Dr. Madigan brings to Syndax over 20 years of clinical hematology expertise and broad experience in the design and execution of early to late-stage clinical programs across oncology and rare diseases.
In February 2022, the Company announced the transition of Michael A. Metzger to the role of Chief Executive Officer and Briggs W. Morrison, M.D., to President, Head of Research and Development. Mr. Metzger and Dr. Morrison, who both serve on the Company’s Board of Directors, joined Syndax together in 2015.
Fourth Quarter 2021 Financial Results

As of December 31, 2021, Syndax had cash, cash equivalents and short-term investments of $439.9 million and 59.0 million shares and share equivalents issued and outstanding. This includes 4.0 million pre-funded warrants.

Fourth quarter 2021 research and development expenses increased to $23.9 million from $15.5 million, and for the full year increased to $88.2 million compared to $50.4 million for 2020. The fourth quarter and full year increases were primarily due to increased clinical trial and CMC activities.

General and administrative expenses for the fourth quarter 2021 increased to $6.9 million from $4.7 million, and, for the year ended December 31, 2021, increased to $25.2 million compared to $22.5 million for the prior year. The fourth quarter and full year increases were primarily due to increased professional fees and employee related expenses.

License revenue for the fourth quarter 2021 increased to $126.6 million from $0.4 million, and, for the year ended December 31, 2021, increased to $139.7 million compared to $1.5 million for the prior year due to revenue related to the license and collaboration agreement with Incyte and the termination of the Company’s license agreement with KKC.

For the three months ended December 31, 2021, Syndax reported a net profit attributable to common stockholders of $96.2 million or $1.81 per share compared to a net loss attributable to common stockholder of $20.4 million or $0.44 per share for the prior year period. For the year ended December 31, 2021, Syndax reported a net profit attributable to common stockholders of $24.9 million or $0.48 per share, compared to a net loss attributable to common stockholders of $77.8 million or $1.87 per share for the prior year.

Financial Update and Guidance

In December 2021, Syndax issued 4,945,000 shares of its common stock and pre-funded warrants to purchase shares of its common stock at approximately $17.50 per share. As a result, Syndax received gross proceeds of approximately $86.5 million. Syndax also issued 1,421,523 shares of its common stock in connection with the Share Purchase Agreement with Incyte Pharmaceuticals at a 30% premium to market for proceeds of $35.0 million.

For the first quarter of 2022, research and development expenses are expected to be $30 to $35 million, and total operating expenses are expected to be $38 to $42 million. For the full year of 2022, research and development expenses are expected to be $130 to $140 million, and total operating expenses are expected to be $160 to $170 million. This does not include any potential cost offsets due to the Incyte collaboration.

Conference Call and Webcast

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 today, Tuesday, March 1, 2022.

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:

Conference ID: 4019975
Domestic Dial-in Number: (855) 251-6663
International Dial-in Number: (281) 542-4259
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.

On March 1, 2022 INOVIO (NASDAQ:INO), a biotechnology company focused on developing and commercializing DNA medicines to help protect people from infectious diseases and help treat people with cancer and HPV-associated diseases, reported financial results for the quarter and year ended December 31, 2021 (Press release, Inovio, MAR 1, 2022, View Source [SID1234609274]). INOVIO’s management will host a live conference call and webcast at 4:30 p.m. Eastern Time today to discuss financial results and provide a general business update. The live webcast and replay may be accessed by visiting INOVIO’s website at View Source

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Dr. J. Joseph Kim, President and CEO of INOVIO, said, "COVID-19 represents a continued and persistent threat to the health of our global community. The highly transmissible Omicron variant underscores the ongoing challenge to improve access to both primary vaccines and boosters. In in vitrotesting we observed that INO-4800 maintained robust T cell responses against the Omicron variant, even though we saw significantly decreased levels of both neutralizing and binding antibodies against Omicron, consistent with the vaccines of other developers. Based on these preserved T cell responses, INOVIO plans to seek approval to amend the primary endpoint of the INNOVATE Phase 3 trial to prevention of severe disease. INOVIO is also evaluating the feasibility of conducting an additional heterologous boost trial for INO-4800, which, if undertaken, would complement the booster trial underway with INOVIO’s partner Advaccine in China."

"In parallel with our COVID-19 efforts, we are also pleased with the clinical progress to date across our DNA medicines platform, including our therapeutic programs in HPV-associated diseases. In the fourth quarter, we completed enrollment of REVEAL2, our second global Phase 3 clinical trial of VGX-3100 for cervical pre-cancer. In addition, we recently completed enrollment in our Phase 1/2 clinical trial for INO-3107, our DNA immunotherapy for the rare disease recurrent respiratory papillomatosis (RRP), which received orphan drug designation from the U.S. FDA. We have also completed enrollment in a Phase 1b trial for Lassa fever, a Phase 1b trial for an Ebola vaccine booster, as well as the first part of our Phase 2 trial for Middle East Respiratory Syndrome (MERS), showcasing the depth and breadth of our DNA medicines pipeline to support global public health efforts against potential pandemic threats," said Dr. Kim.

INOVIO 4Q21 and Year-End 2021 Highlights:

INO-4800 was observed to provide full maintenance of T cell responses, but significantly decreased levels of antibodies against the Omicron variant in line with results observed with other COVID-19 vaccines.
INOVIO plans to seek regulatory approval to amend the primary endpoint of the INNOVATE Phase 3 trial from prevention of virologically confirmed COVID-19 disease to prevention of severe disease due to COVID-19. INOVIO has paused enrollment in INNOVATE in preparation for this potential change.
The Company is evaluating the feasibility of pursuing an INOVIO-sponsored heterologous boost non-inferiority clinical trial with INO-4800 compared to viral vector and inactivated COVID-19 vaccines.
INOVIO’s partner Advaccine completed enrollment of its 200-participant homologous and 267-participant heterologous boost trial with INO-4800 in China.
INO-4800 was selected by the World Health Organization (WHO) for inclusion in a global Phase 3 trial for COVID-19 (Solidarity Trial Vaccines).
REVEAL2, the second of two Phase 3 trials for VGX-3100 for cervical high-grade squamous intraepithelial lesions (HSIL), completed enrollment, with top-line efficacy and safety data anticipated in the second half of 2022.
INOVIO completed enrollment of its Phase 1/2 trial of INO-3107 for RRP.
Enrollment completed for the Phase 1b heterologous booster clinical trial of INO-4201 as a potential Ebola booster vaccine in collaboration with GuardRX and Geneva University Hospitals, an effort funded by the Defense Advanced Research Projects Agency (DARPA).
Fourth Quarter and Year-End Program Updates

Infectious Diseases

INO-4800: COVID-19

INO-4800 Omicron Assessment

INOVIO conducted an in vitroassessment of the cross-reactivity of INO-4800 vaccine-induced immune responses against the Omicron variant of SARS-CoV-2. Testing demonstrated a maintenance of T cell responses, including CD8+ responses, but as seen with the vaccines of other developers, significantly decreased levels of both neutralizing and binding antibodies against Omicron.

INOVIO observed full maintenance of T cell responses against the Omicron variant in clinical samples from participants who had received INO-4800. The maintenance and preservation of T cell responses continue to remain a consistent observation for INO-4800 against the ancestral COVID-19 virus and across all variants of concern (VOCs) tested to date, including Omicron. The Company believes this is a critical observation as T cell responses are thought to play an important role1 in protection against severe disease2 and death and may be central to the durability of vaccine protection.

INO-4800 in INNOVATE Phase 3 Trial

In response to the dominance of the Omicron variant globally and the persistence of cross-reactive T cell responses generated by INO-4800, including CD8+, across all variants of concern to date, INOVIO plans to seek regulatory approval to amend the primary endpoint of INNOVATE from prevention of virologically confirmed COVID-19 disease to prevention of severe disease due to COVID-19. The Company believes INO-4800’s ability to generate T cell responses could be critical in meeting the proposed amended primary endpoint.

In addition, to reflect the potential impact of the Omicron variant on INNOVATE, the Data Safety Monitoring Board (DSMB) recommended that INOVIO pause enrollment of new participants in the global Phase 3 clinical trial of INO-4800 in order to update the Informed Consent Form and Investigator Brochure. As a result of the DSMB’s recommendation, as well as the company’s plans to seek approval to amend the trial’s primary endpoint, INOVIO has paused enrollment of new participants in INNOVATE. Interim efficacy data from INNOVATE will therefore not be available in the first half of 2022 as previously expected.

Heterologous and Homologous Boost Trials

INOVIO’s partner Advaccine has completed enrollment of its 200-participant homologous and 267-participant heterologous boost trials in China. The trials are designed to evaluate safety, tolerability, and immunogenicity of INO-4800 as a homologous boost where INO-4800 was administered as the primary vaccine and as a heterologous boost in which inactivated vaccines were administered as the primary vaccine.

INOVIO is evaluating the feasibility of an additional ex-US heterologous boost (vaccination with a different vaccine from the primary series) trial with INO-4800 as a booster in a non-inferiority clinical trial compared to viral vector and inactivated COVID-19 vaccines. Currently licensed vaccines may not meet the global demand for boosters to address waning protection from these primary vaccinations, a need which some regulatory agencies are considering with respect to evaluating clinical pathways for heterologous boost vaccines. Key features of INOVIO’s DNA vaccine technology that make it a potentially favorable primary and booster candidate include generating T-cell responses against multiple variants of concern, lack of anti-vector immunity, tolerability of re-administration, and thermostability for transport, storage, and distribution.

Solidarity Trial Vaccines

INO-4800 is one of two COVID-19 vaccine candidates currently being tested in a large, international, randomized controlled Phase 3 clinical trial, called the Solidarity Trial Vaccines, being funded, sponsored, and conducted by the World Health Organization (WHO). INO-4800 was selected for inclusion in the Solidarity Trial Vaccines by the WHO’s independent vaccine prioritization advisory group.

INO-4500: Lassa Fever

In October 2021 INOVIO announced that the Phase 1b clinical trial for INO-4500, its DNA vaccine candidate for Lassa fever, completed full enrollment of 220 participants. This Phase 1b trial (LSV-002 – NCT04093076), which is funded by Coalition for Epidemic Preparedness Innovations (CEPI) is ongoing at the Noguchi Memorial Institute for Medical Research in Accra, Ghana, and is the first vaccine clinical trial for Lassa fever conducted in West Africa, where the viral illness is endemic. The dosing regimen involved two intradermal vaccinations at 0 and 28 days with either 1.0 mg or 2.0 mg doses. In addition to providing insights on the INO-4500 safety and immunogenicity profile, this trial will inform dose selection for subsequent Phase 2 trials in West Africa.

INO-4700: Middle East Respiratory Syndrome (MERS)

INOVIO has dosed and completed enrollment for the first part (dose finding stage) of the Phase 2 trial (192 participants) of INO-4700 against Middle East Respiratory Syndrome (MERS), a disease in the coronavirus family for which there are no approved vaccines.

The multi-center Phase 2 trial is a randomized, double-blinded, placebo-controlled trial designed to evaluate the safety, tolerability, and immunogenicity of INO-4700 in approximately 500 healthy adult participants. The trial, which is sponsored by INOVIO and fully funded by the CEPI, is being conducted at sites in Jordan, Lebanon, and Kenya, where MERS cases have been reported.

INO-4201: Ebola

Enrollment of 46 healthy participants has been completed as part of a randomized, placebo-controlled, Phase 1b clinical trial evaluating the safety, tolerability, and immunogenicity of INOVIO’s Ebola vaccine candidate INO-4201. The trial (NCT04906629) titled "Phase 1b, Placebo-controlled Randomized Clinical Trial to Evaluate the Safety, Tolerability and Immunogenicity of INO-4201 Followed by Electroporation as a Booster Vaccination in Healthy Volunteers Who Have Previously Received the VSV-ZEBOV Vaccine" will assess whether INO-4201 can be used as a booster in participants previously vaccinated with rVSV-ZEBOV (Ervebo). Ervebo is a replication-competent, live, attenuated recombinant vesicular stomatitis virus (rVSV)-based vaccine approved by the U.S. FDA for the prevention of disease caused by Zaire ebolavirus in individuals 18 years of age and older as a single-dose administration.

HPV-Associated Diseases

VGX-3100: Cervical HSIL (REVEAL1 / REVEAL2)

INOVIO completed enrollment in REVEAL2, its second global Phase 3 clinical trial of VGX-3100 for cervical HSIL. Top-line efficacy and safety data are expected to be available in the second half of 2022.

INOVIO completed the 52-week safety follow-up of participants in REVEAL1 and showed that VGX-3100 remained well-tolerated through Week 88. In addition, participants treated with VGX-3100 who met the primary endpoint at Week 36 remained clear of HPV-16 and/or HPV-18 at Week 88.

Additionally, INOVIO is pleased with progress to date with partner QIAGEN on the co-development of a liquid biopsy-based diagnostic product based on next-generation sequencing (NGS) technology to guide clinical decision-making for the use of VGX-3100 in cervical HSIL. This biomarker, if validated, may have the potential to identify those women who are more likely to have a favorable treatment outcome, specifically the regression of cervical HSIL and clearance of virus. INOVIO anticipates having additional information on its biomarker development progress in 2022.

INO-3107: Recurrent Respiratory Papillomatosis (RRP)

Subsequent to year end, the company completed enrollment of 32 participants in its open-label, multicenter Phase 1/2 clinical trial with INO-3107 in participants with HPV 6 and/or 11-associated RRP. RRP is a rare disease (estimated at 15,000 active cases in the U.S.) that is characterized by the growth of tumors in the respiratory tract caused by the human papillomavirus. The first participant was dosed in November 2020. All 32 adult participants first underwent surgical removal of their papilloma(s) and then received four doses of INO-3107, one every three weeks. The trial is assessing safety, tolerability, immunogenicity, and efficacy of INO-3107. The company expects preliminary efficacy data from a portion of participants from this Phase 1/2 trial in the second half of this year.

INO-3107 previously received orphan drug indication from the U.S. FDA in July 2020.

Immuno-oncology

INO-5401: Newly Diagnosed Glioblastoma Multiforme (GBM)

In November 2021, Dr. David Reardon, Clinical Director of the Center for Neuro-Oncology at Dana-Farber Cancer Institute and the Coordinating Principal Investigator, presented updated data from the GBM-001 Phase 2 trial at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) pre-conference workshop. Overall survival at 24 months was 22% (7/32) for the MGMT unmethylated cohort and 55% (11/20) for the MGMT methylated cohort. The trial showed that INO-5401+INO-9012 with cemiplimab and radiation/TMZ have an acceptable safety profile, are immunogenic, and may improve survival in newly diagnosed GBM. We look forward to continuing our investigations into GBM and other cancers.

Manufacturing

In October 2021, INOVIO signed a non-binding MOU with Colombia’s Ministry of Health and Social Protection reflecting the intent to advance efforts to combat the pandemic and endemic threat posed by COVID-19 and to better prepare for future public health emergencies. The MOU creates a framework for collaboration by which INOVIO and the government can explore knowledge sharing, technology licensing, and capacity building that support developing and producing vaccines and other biopharmaceuticals in Colombia. The potential results of these efforts include developing local manufacturing capabilities for INOVIO’s DNA medicines and related products and technologies.

Fourth Quarter and Full Year 2021 Financial Results

Total revenue was $839,000 and $1.8 million for the quarter and year-ended December 31, 2021, respectively, compared to $5.6 million and $7.4 million for the respective periods in 2020. Total operating expenses were $106.3 million and $303.0 million for the quarter and year-ended December 31, 2021, respectively, compared to $34.9 million and $131.5 million for the respective periods in 2020.

INOVIO’s net loss for the quarter and year ended December 31, 2021 was $106.9 million, or $0.50 per basic and diluted share, and $303.7 million, or $1.45 per basic and diluted share, respectively, compared to net loss of $24.3 million, or $0.14 per basic and diluted share, and $166.4 million, or $1.07 per basic and diluted share, for the quarter and year ended December 31, 2020, respectively.

Operating Expenses

Research and development (R&D) expenses for the quarter and year-ended December 31, 2021, were $92.3 million and $249.2 million, respectively, compared to $26.3 million and $94.2 million for the respective periods in 2020. The year-over-year increase in R&D expenses was primarily related to higher drug manufacturing, outside services and clinical trial expenses related to INO-4800, expenses related to the acquisition and installation of manufacturing equipment related to INO-4800, higher engineering services, expensed equipment and inventory related to our CELLECTRA 3PSP device array automation project, and higher employee and contractor compensation, among other variances.

General and administrative (G&A) expenses were $14.0 million and $53.8 million, respectively, for the quarter and year ended December 31, 2021, versus $8.6 million and $37.2 million, respectively, for the same periods in 2020. The year-over-year increase in G&A expenses was primarily related to an increase in employee compensation, including non-cash stock-based compensation, due to an increase in employee headcount, among other variances.

Capital Resources

As of December 31, 2021, cash and cash equivalents and short-term investments were $401.3 million compared to $411.6 million as of December 31, 2020. As of December 31, 2021, the Company had 217.4 million common shares outstanding and 234.0 million common shares outstanding on a fully diluted basis, after giving effect to the exercise, vesting and conversion, as applicable, of its outstanding options, restricted stock units, convertible preferred stock, and convertible debt.

INOVIO’s balance sheet and statement of operations are provided below. Additional information is included in INOVIO’s annual report on Form 10-K for the year ended December 31, 2021, which can be accessed at: View Source

Conference Call / Webcast Information

INOVIO’s management will host a live conference call and webcast at 4:30 p.m. Eastern Time today to discuss INOVIO’s financial results and provide a general business update. The live webcast and replay may be accessed by visiting INOVIO’s website at View Source

References

Noh, J.Y., Jeong, H.W., Kim, J.H. et al. T cell-oriented strategies for controlling the COVID-19 pandemic. Nat Rev Immunol21, 687–688 (2021).
Rydyznski Moderbacher, C. et al. Antigen-specific adaptive immunity to SARS-CoV-2 in acute COVID-19 and associations with age and disease severity. Cell 183, 996–1012 (2020).

On March 1, 2022 Avid Bioservices, Inc. (NASDAQ:CDMO), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality services to biotechnology and pharmaceutical companies, reported that it will report financial results for the third quarter of fiscal year 2022 on March 8, 2022 after market close and will host a conference call and webcast at 1:30 PM Pacific Time (4:30 PM Eastern Time) (Press release, Avid Bioservices, MAR 1, 2022, View Source [SID1234609273]). Members of Avid’s senior management will discuss financial results for the third quarter and review recent corporate developments.

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To listen to the live webcast, or access the archived webcast, please visit: View Source

To listen to the conference call, please dial (877) 312-5443 or (253) 237-1126 and request the Avid Bioservices call.

On March 1, 2022 Allakos Inc. (the "Company") (Nasdaq: ALLK), a biotechnology company developing lirentelimab (AK002) and AK006 for the treatment of allergic and inflammatory diseases, reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Allakos, MAR 1, 2022, View Source [SID1234609272]).

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Recent Events

Initiated a Phase 2 randomized, double-blind, placebo-controlled study of subcutaneous lirentelimab in patients with moderate-to-severe atopic dermatitis in the fourth quarter of 2021.
Reported topline data from ENIGMA 2, a Phase 3 randomized, double-blind, placebo-controlled study of lirentelimab in patients with eosinophilic gastritis/eosinophilic duodenitis ("EG"/"EoD") in the fourth quarter of 2021.
Reported topline data from KRYPTOS, a Phase 2/3 randomized, double-blind, placebo-controlled study of lirentelimab in patients with eosinophilic esophagitis ("EoE") in the fourth quarter of 2021.
Upcoming Milestones

Hold an End-of-Phase 2 meeting with the FDA during second quarter of 2022 to discuss the Phase 2/3 KRYPTOS data and the development path with subcutaneous lirentelimab in patients with EoE.
Report topline data from the Phase 3 study of lirentelimab in patients with EoD (EoDyssey) in the third quarter of 2022.
Initiate a Phase 2b randomized, double-blind, placebo-controlled study of subcutaneous lirentelimab in patients with chronic spontaneous urticaria in the middle of 2022.
Complete IND-Enabling studies of AK006 during 2022 and initiate the first-in-human study in the first half of 2023.
Fourth Quarter and Full Year 2021 Financial Results

Research and development expenses were $72.9 million in the fourth quarter of 2021 as compared to $28.5 million in the same period in 2020, an increase of $44.4 million. Research and development expenses were $196.3 million for the full year 2021 as compared to $105.5 million in the same period in 2020, an increase of $90.8 million.

General and administrative expenses were $23.2 million in the fourth quarter of 2021 as compared to $15.8 million in the same period in 2020, an increase of $7.4 million. General and administrative expenses were $75.1 million for the full year 2021 as compared to $51.5 million in the same period in 2020, an increase of $23.6 million.

Allakos reported a net loss of $94.4 million in the fourth quarter of 2021 as compared to $44.3 million in the same period in 2020, an increase of $50.1 million. Net loss per basic and diluted share was $1.73 for the fourth quarter of 2021 compared to $0.86 in the same period in 2020. Net loss was $269.9 million for the full year 2021 as compared to $153.5 million in the same period in 2020, an increase of $116.4 million. Net loss per basic and diluted share was $5.01 for the full year 2021 compared to $3.10 in the same period in 2020.

Allakos ended the fourth quarter of 2021 with $424.2 million in cash, cash equivalents and marketable securities.

On March 1, 2022 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported an update on its discussions with the U.S. Food and Drug Administration (FDA) regarding its previously planned supplemental New Drug Application (sNDA) submission based on the data from the Phase 3 SIENDO study evaluating selinexor as a front-line maintenance therapy following chemotherapy in patients with advanced or recurrent endometrial cancer (Press release, Karyopharm, MAR 1, 2022, View Source [SID1234609271]).

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During a productive meeting with the FDA, the Company received feedback that the current SIENDO study top-line results are unlikely to support an sNDA approval. Karyopharm and the FDA participants had differing views on the study significance and overall clinical benefit for the whole population and discussed that further exploration of patients with advanced or recurrent endometrial cancer with p53 wild-type is warranted. The Company will continue to collect and analyze the SIENDO study data and work with the FDA to explore all regulatory pathways for patients with p53 wild-type. In addition, considering the FDA’s feedback, the Company intends to initiate a new placebo-controlled, randomized clinical study of selinexor in patients with p53 wild-type endometrial cancer and believes top-line data will be available in the first half of 2024. Karyopharm plans to rapidly initiate this study of selinexor in patients with p53 wild-type this year, working with the FDA as well as established networks and partners, including the European Network of Gynaecological Oncological Trial groups (ENGOT) and the Gynecologic Oncology Group Foundation, Inc. (GOG-F).

"We strongly believe in selinexor’s potential in patients with p53 wild-type and are excited to further evaluate it in this patient population to better understand its potential to address the unmet need in women with endometrial cancer," said Sharon Shacham, Chief Scientific Officer of Karyopharm. "Given the significant need for new therapeutic options, we have a tremendous sense of urgency to design and enroll a new trial as quickly as possible and believe we are well-positioned to do so working with our existing SIENDO clinical trial sites."

"The majority of endometrial cancers are diagnosed at an early stage and are typically curable with surgery, however, women with advanced and recurrent endometrial cancer have very limited therapeutic options following front-line treatments and prognosis is typically poor," said Professor Ignace Vergote, principal investigator and gynecologist oncologist, ENGOT and the Belgium and Luxembourg Gynaecological Oncology Group (BGOG), University of Leuven, Leuven Cancer Institute, Leuven, Belgium. "Selinexor has the potential to transform the way advanced or recurrent endometrial cancer is treated in patients with p53 wild-type and I look forward to learning more from a new study."

The Phase 3 SIENDO study has been selected for presentation at the upcoming European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper)’s Virtual Plenary taking place on Thursday, March 17, 2022 and at the Society for Gynecologic Oncology 2022 Annual Meeting on Women’s Cancer taking place March 18-21, 2022 in Phoenix, Arizona. Details regarding these presentations, along with an investor conference call to review the SIENDO results, will be announced soon.

About the SIENDO Study

The Phase 3 SIENDO study (ENGOT-EN5/GOG-3055) is a multicenter, blinded, placebo-controlled, randomized study evaluating the efficacy and safety of selinexor as a maintenance therapy following chemotherapy in patients with advanced or recurrent endometrial cancer. The study enrolled 263 patients with primary stage IV or recurrent disease who had a partial or complete response after at least 12 weeks of standard taxane-platinum combination chemotherapy. Patients were randomized 2:1 to receive either maintenance therapy of 80mg of selinexor taken once weekly, or placebo, until disease progression. The primary endpoint of the study is statistically significant improvement of progression-free survival compared to placebo. The goal of the study was to demonstrate a hazard ratio of 0.6 or better. In partnership with Karyopharm, the study was initiated by the ENGOT group. In the U.S., the collaboration includes the GOG-F.

About Endometrial Cancer

Endometrial cancer is the most common cancer of the female reproductive organs in the U.S., with approximately 66,000 new cases expected in 2022 leading to nearly 13,000 deaths.1 In 2020, there were approximately 130,000 new cases and 29,000 deaths in Europe from endometrial cancer, while on a global scale there were 417,000 new cases and approximately 97,000 deaths.2 More than 90 percent of uterine body cancers occur in the endometrium, so the actual numbers for endometrial carcinoma cases and deaths are slightly lower than these estimates, which include both endometrial carcinomas and uterine sarcomas. Unlike other cancers that have decreased with preventative measures, endometrial cancer is on the rise.3 Risk factors include obesity, Type 2 diabetes, high-fat diets, use of tamoxifen and oral estrogens, and delayed menopause.4 While the majority of endometrial cancers are diagnosed at early stages, approximately 14,000 patients in the U.S. are diagnosed with advanced disease that cannot be fully removed using surgery.5 These patients, and those with recurrent disease, are treated with chemotherapy. Chemotherapy does not cure patients with endometrial cancer. The use of later lines of chemotherapy are intended to control symptoms rather than cure the disease. There are no approved therapies in the maintenance setting for patients with advanced or recurrent endometrial cancer. The current standard of care is a "watch and wait" approach.6

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm’s Selective Inhibitor of Nuclear Export (SINE) compounds to be approved for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with Velcade (bortezomib) and dexamethasone (XVd) in patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in patients with heavily pre-treated multiple myeloma; and (iii) in patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in a growing number of ex-U.S. territories and countries, including Europe, the United Kingdom, China, South Korea and Israel, and is marketed in those areas by Karyopharm’s global partners. Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including myelofibrosis. For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at:

Tel: +1 (888) 209-9326
Email: [email protected]

XPOVIO (selinexor) is a prescription medicine approved:

In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd).
In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti–CD38 monoclonal antibody (Xd).
For the treatment of adult patients with relapsed or refractory diffuse large B–cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.
Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony–stimulating factors.
Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.
Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.
Serious Infection: Monitor for infection and treat promptly.
Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.
Embryo–Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.
Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3–4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.
The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.
The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3–4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions were infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.
Use In Specific Populations

Lactation: Advise not to breastfeed.

For additional product information, including full prescribing information, please visit www.XPOVIO.com.