On May 9, 2022 BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a commercial-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, reported its financial results for the first quarter ended March 31, 2022 and provided an update on key strategic initiatives (Press release, BioXcel, MAY 9, 2022, View Source [SID1234613879]).

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"2022 has already been a transformative year during which we have made monumental progress on our journey to become the leading AI-enabled neuroscience company. Our recent achievements across our clinical, business, and commercial priorities, highlighted by our first FDA approval of IGALMI, have positioned us to realize our 2022 goals and beyond," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "Building on this momentum, we are excited to launch IGALMI, advance our proven technology, and continue delivering innovative medicines to patients and caregivers."

Company Highlights

Neuroscience Franchise

IGALMI Approval and Commercial Launch

IGALMI was approved by the FDA on April 5, 2022 for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults.1 Within these two patient populations in the U.S., up to 25 million agitation episodes occur each year.2-4

The Company is focused on the following key commercial launch activities to provide access to patients and caregivers:

·Institutional Sales Force: Seasoned sales specialists were onboarded, undergoing training, and will be deployed for launch. The team has an average of over 21 years of industry experience, 14 years in hospital settings, and 8 product launches.
oLaunch meeting to be held in mid-May in preparation for national salesforce deployment on May 23rd across priority regions and customers.
·Market Access Team: Currently engaging with group purchasing organizations (GPOs) and high value Integrated Delivery Networks (IDNs).
·Trade Launch: Product load into distribution channels in Q3 2022.

Clinical Pipeline

BXCL501, a proprietary, sublingual film formulation of dexmedetomidine, is being investigated in multiple neuropsychiatric conditions. BXCL501 has received Breakthrough Therapy and Fast Track designation for the acute treatment of agitation associated with dementia.

Indication Expansion

·Alzheimer’s Disease-related Agitation: TRANQUILITY program designed to maximize BXCL501 opportunity to treat Alzheimer’s Disease-related agitation. There are an estimated 100 million agitation episodes in Alzheimer’s patients occurring in the U.S. annually.5
oTRANQUILITY II: First patient dosed; top-line data readout in Q4 2022/early Q1 2023.
oTRANQUILITY III: Trial underway with enrollment initiating in 2H 2022.
oSelection of 40 mcg and 60 mcg dosing regimens in pivotal trial supported by breakthrough designation from the FDA, along with positive efficacy, safety, and tolerability data observed in 100 patients in the Phase 1b/2 TRANQUILITY trial and a recent study of BXCL501 40 mcg.

·Adjunctive Treatment for Major Depressive Disorder (MDD): Initiated Phase 1 multiple ascending dose trial in healthy volunteers.

Geographic Expansion

·Marketing Authorization Application: Submission to the European Medicines Agency (EMA) for BXCL501 for acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults in 2Q 2022.
·Japan Entry: Company exploring market entry strategies to expand access of IGALMI to patients in Japan.

OnkosXcel Therapeutics

Established OnkosXcel Therapeutics as a wholly owned subsidiary to focus on the sustained expansion and optimization of the Company’s immuno-oncology (I-O) franchise, including its most advanced I-O program, BXCL701. BXCL701 is an investigational, orally administered, systemic innate immune activator in development for the treatment of aggressive forms of prostate cancer and advanced solid tumors that are refractory or treatment naïve to checkpoint inhibitors.

·Metastatic Castration-Resistant Prostate Cancer (mCRPC) Program: Following positive Phase 2 data for BXCL701 in combination with KEYTRUDA (pembrolizumab) announced this past February, continued ongoing Phase 2 trial in mCRPC patients with either small cell neuroendocrine carcinoma (SCNC) or adenocarcinoma phenotype.
oExpect to complete enrollment of 28-patient SCNC cohort in 2H 2022.
oFirst patient enrolled in adenocarcinoma randomized trial expansion evaluating BXCL701 monotherapy vs. BXCL701-KEYTRUDA combination therapy.

·Solid Tumors Program (Checkpoint Naïve and Refractory): Additional efficacy data from MD Anderson-led open-label Phase 2 basket trial of BXCL701 and KEYTRUDA is expected in 2H 2022.

Strategic Financing

·Announced $260 Million Strategic Financing with Oaktree and Qatar Investment Authority: Full execution of this financing would extend the Company’s cash runway into 2025 to support the commercial launch of IGALMI and achieve key clinical and regulatory milestones.

First Quarter 2022 Financial Results

Research and Development Expenses: Research and development expenses were $18.6 million for the first quarter of 2022, compared to $14.7 million for the same period in 2021. The increased expenses were primarily attributable to clinical trial costs related to the Company’s TRANQUILITY program.

General and Administrative Expenses: General and administrative expenses were $12.9 million for the first quarter of 2022, as compared to $11.6 million for the same period in 2021. The increase was primarily due to personnel and costs related to the commercial launch readiness efforts for IGALMI in the U.S.

Net Loss: BioXcel Therapeutics reported a net loss of $31.5 million for the first quarter of 2022, compared to a net loss of $26.4 million for the same period in 2021.

As of March 31, 2022, cash and cash equivalents totaled approximately $200 million. This excludes contributions from the $260 million strategic financing announced in April. To date the Company has drawn $70M of the loan agreement and has met the milestone to receive $30M of the royalty financing which is expected to be drawn in the second quarter of 2022.

Conference Call

BioXcel Therapeutics will host a conference call and webcast May 9, 2022, at 8:30 a.m., ET, to discuss its first quarter 2022 financial results and provide an update on recent operational highlights. To access the call, please dial 877-407-5795 (domestic) and 201-689-8722 (international). A live webcast of the call will be available on the Investors section of the BioXcel website, www.bioxceltherapeutics.com, and a replay of the call will be available through August 9, 2022.

BioXcel Therapeutics may use its website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com. In addition, you may automatically receive email alerts and other information about the Company when you enroll your email address by visiting the "Email Alerts" option under the News/Events menu of the Investors & Media section of its website.

About TRANQUILITY II and III

Initiated in December of 2021, TRANQUILITY II and III are pivotal Phase 3 trials evaluating BXCL501 for the acute treatment of agitation in patients with Alzheimer’s disease. The trials expand the evaluation of patients who experience agitation across diverse medical settings and across the range of dementia severity. TRANQUILITY II and III are designed to maximize the opportunity of BXCL501 for the treatment of the full spectrum of agitation associated with AD. Each trial will enroll approximately 150 dementia patients 65 years and older who will self-administer 40 mcg or 60 mcg of BXCL501 or placebo whenever agitation episodes occur over a three-month period. TRANQUILITY II will assess patients in assisted living or residential facilities requiring minimal assistance with activities of daily living. TRANQUILITY III will assess patients residing in nursing homes with moderate to severe dementia and require moderate or greater assistance with activities of daily living. The studies will assess agitation as measured by the changes from baseline in the Positive and Negative Syndrome Scale-Excitatory Component (PEC) and Pittsburgh Agitation Scale (PAS) total scores. The primary efficacy endpoint for both studies is change in PEC score from baseline measured at two hours after the initial dose and subsequent doses.

On May 9, 2022 FibroGen, Inc. (NASDAQ: FGEN) reported financial results for the first quarter 2022 and provided an update on the company’s recent developments (Press release, FibroGen, MAY 9, 2022, View Source [SID1234613912]).

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"We continue our progress in advancing pamrevlumab in three high value indications and are delighted to have completed enrollment of the ZEPHYRUS-1 Phase 3 study in idiopathic pulmonary fibrosis," said Enrique Conterno, Chief Executive Officer, FibroGen. "In addition, roxadustat is off to a strong start in 2022 in China with significant year over year volume growth."

Recent Developments:

Completed enrollment of the ZEPHYRUS-1 Phase 3 clinical trial of pamrevlumab in patients with idiopathic pulmonary fibrosis (IPF).
Completed enrollment of the LELANTOS-1 Phase 3 clinical trial of pamrevlumab in non-ambulatory patients with Duchenne muscular dystrophy (DMD).
Our partner Astellas received approval for roxadustat in Russia for the treatment of adult patients with symptomatic anemia associated with chronic kidney disease (CKD), which triggered a $25 million milestone payable to FibroGen.
China Performance:

FibroGen’s net product revenue under U.S. GAAP from sale of roxadustat in China was $18.9 million compared to $15.4 million in the first quarter of 2021. Increase driven mainly due to release of deferred revenue.
First quarter total roxadustat net sales in China1 by FibroGen and the distribution entity (JDE) jointly owned by FibroGen and AstraZeneca was $43.5 million, flat as compared to the first quarter of 2021. This result was driven by an increase in volume of over 70% offset by the recent National Reimbursement Drug List (NRDL) price reduction.
Roxadustat continues to be the number one brand based on value share in the anemia of CKD market in China.
Upcoming Milestones:

Interim analysis of event free survival of the LAPIS Phase 3 study of pamrevlumab in locally advanced pancreatic cancer (LAPC) to be conducted in 2Q 2022.
Topline data from the LELANTOS-1 Phase 3 study of pamrevlumab in DMD expected 1H 2023.
Expect to complete enrollment in the LELANTOS-2 Phase 3 study of pamrevlumab in ambulatory patients with DMD in 2Q 2022.
Topline data from the ZEPHYRUS-1 Phase 3 study of pamrevlumab in IPF expected mid-2023.
Topline data from the MATTERHORN Phase 3 study of roxadustat in anemia of myelodysplastic syndromes (MDS) expected 1H 2023.
Financial:

Total revenue for the first quarter of 2022 was $60.8 million, as compared to $38.4 million for the first quarter of 2021.
Net loss for the first quarter of 2022 was $63.2 million, or $0.68 net loss per basic and diluted share, compared to a net loss of $71.8 million, or $0.78 net loss per basic and diluted share one year ago.
At March 31, 2022, FibroGen had $565.4 million in cash – defined as cash, cash equivalents, investments, and accounts receivable.
Based on our latest forecast, we estimate a 2022 ending cash balance of $310-340 million.
________________
1 Total roxadustat net sales in China includes sales made by the distribution entity as well as FibroGen China’s direct sales, each to its own distributors. The distribution entity jointly owned by AstraZeneca and FibroGen is not consolidated into FibroGen’s financial statements.

Conference Call and Webcast Details
FibroGen will host a conference call and webcast today, Monday, May 9, 2022, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time) to discuss financial results and provide a business update. A live audio webcast of the call may be accessed in the investor section of the Company’s website, www.fibrogen.com. To participate in the conference call by telephone, please dial 1 (877) 658-9081 (U.S. and Canada) or 1 (602) 563-8732 (international), reference the FibroGen first quarter 2022 financial results conference call, and use confirmation number 2487763. A replay of the webcast will be available shortly after the call for a period of 7 days. To access the replay, please dial 1 (855) 859-2056 (domestic) or 1 (404) 537-3406 (international) and use passcode 2487763.

About Pamrevlumab
Pamrevlumab is a potential first-in-class antibody being developed by FibroGen that inhibits the activity of connective tissue growth factor (CTGF), an important biological mediator in fibrotic and proliferative disorders. Pamrevlumab is in Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF), locally advanced unresectable pancreatic cancer (LAPC), and Duchenne muscular dystrophy (DMD). Pamrevlumab is an investigational drug and not approved for marketing by any regulatory authority. For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.

About Roxadustat
Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin. Roxadustat is in clinical development for anemia of chronic kidney disease (CKD) and anemia associated with myelodysplastic syndromes (MDS), and for chemotherapy-induced anemia (CIA).

Roxadustat is approved in European Union (EU) member states, including the European Economic Area (EEA) countries, as well as in Japan, China, Chile, South Korea, Russia, and the UK for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). Several other licensing applications for roxadustat have been submitted by partners, Astellas and AstraZeneca to regulatory authorities across the globe, and are currently under review.

Astellas and FibroGen are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in territories including Japan, Europe, Turkey, Russia and the Commonwealth of Independent States, the Middle East, and South Africa. FibroGen and AstraZeneca are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in the U.S., China, other markets not licensed to Astellas.

On May 9, 2022 MaxCyte, Inc., (NASDAQ: MXCT; LSE: MXCT), a leading commercial cell-engineering company focused on providing enabling platform technologies to advance innovative cell-based research as well as next-generation cell therapeutic discovery, development and commercialization, reported financial results for the first quarter ended March 31, 2022 and increased full year 2022 revenue guidance (Press release, MaxCyte, MAY 9, 2022, View Source [SID1234613928]).

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First Quarter Highlights

Total revenue of $11.6 million in the first quarter of 2022, an increase of 78% over the first quarter of 2021 driven by strong growth in the core business; core business revenues grew 48% consisting of revenue from cell therapy customers increasing by 57% and drug discovery customers by 23%.
Generated a total of $2.0 million in Strategic Platform License (SPL) Program-related revenue in the first quarter of 2022, compared to immaterial SPL Program-related revenue in the first quarter of 2021.
2022 revenue guidance includes expectations for core business revenue growth to be at least 25% and expected SPL Program-related revenue of approximately $4 million.
With the addition of Intima Bioscience in February 2022, the total number of SPLs now stands at 16.
"We are pleased with this positive start to 2022 at MaxCyte, with very strong first quarter results, including 48% year-over-year core business revenue growth driven by ongoing significant growth in sales to cell therapy customers. We are encouraged by the continued expansion of our portfolio of SPLs with the addition of Intima Bioscience, our sixteenth SPL, as well as the exciting clinical progress of our existing SPL partners. The milestone revenue recorded over the period reflects the progress being made by our partners in early and mid-stage clinical development programs," said Doug Doerfler, President and CEO of MaxCyte.

"I am proud of our continued support for the clinical progress of our partners and the success of our growing global commercial team.

"In addition to the progress made by SPL programs that have entered the clinic, our SPL partners are using MaxCyte’s technology to work on a broad range of new cell types, approaches and indications including solid tumors and autoimmune disease, which also demonstrates the depth and breadth of our ExPERT platform. Ongoing investments in our field and lab science teams and the progress of our in-house manufacturing initiative leaves us well-positioned to support growing adoption of the ExPERT platform technology for cellular-based research and next-generation therapeutic development."

The following table provides details regarding the sources of our revenue for the periods presented.

Total revenue for the first quarter of 2022 was $11.6 million, compared to $6.5 million in the first quarter of 2021, representing growth of 78%.

Core business revenue was $9.6 million, including revenue growth from cell therapy customers of 57% and from drug discovery customers of 23%, compared to core business revenue of $6.5 million in the same period last year.

Our SPL Program-related revenue was $2.0 million, compared to immaterial SPL Program-related revenue in the first quarter of 2021.

Gross profit for the first quarter of 2022 was $10.5 million (91% gross margin), compared to $5.8 million (89% gross margin) in the same period of the prior year. The increase in gross margin was driven by the higher SPL Program-related revenues; excluding SPL Program-related revenues, gross margin was relatively unchanged.

Operating expenses for the first quarter of 2022 were $14.7 million, compared to operating expenses of $12.2 million in the first quarter of 2021. The prior year operating expenses included $3.9 million of CARMA-related expenses that did not recur in 2022, as we have ceased developing the CARMA platform. The overall increase in operating expenses was primarily driven by increased headcount to support growth in field sales and science, manufacturing and lab teams. Growth in public company-related and stock-based compensation expense also contributed to the higher level of expenses compared with the same period a year ago.

First quarter 2022 net loss was $4.1 million compared to net loss of $7.1 million for the same period in 2021; EBITDA, a non-GAAP measure, was a loss of $3.7 million for the first quarter of 2022, compared to a loss of $6.4 million for the first quarter of the prior year; stock-based compensation expense was $2.5 million versus $1.3 million for the same period in the prior year.

Total cash, cash equivalents and short-term investments were $246.3 million as of March 31, 2022.

2022 Revenue Guidance

Management is increasing 2022 revenue guidance based on our expectations for the core business.

We expect core business revenue (instruments and disposables to cell therapy and drug discovery customers and excluding program-related revenue) to grow at least 25% compared to 2021 core business revenue. We also continue to expect SPL Program-related revenue to be approximately $4 million in 2022.

Webcast and Conference Call Details

MaxCyte will host a conference call today, May 9, 2022, at 4:30 p.m. Eastern Time. Interested parties may access the live teleconference by dialing (844) 679-0933 for domestic callers, (918) 922-6914 for international callers, for 0203 1070 289 U.K domestic callers, or for 0800 0288 438 U.K. international callers followed by Conference ID: 1953037. A live and archived webcast of the event will be available on the "Events" section of the MaxCyte website at View Source

Non-GAAP Financial Measures

This press release contains EBITDA, which is a non-GAAP measure defined as earnings, before interest, tax, depreciation and amortization. MaxCyte believes that EBITDA provides useful information to management and investors relating to its results of operations. The company’s management uses this non-GAAP measure to compare the company’s performance to that of prior periods for trend analyses, and for budgeting and planning purposes. The company believes that the use of EBITDA provides an additional tool for investors to use in evaluating ongoing operating results and trends and in comparing the company’s financial measures with other companies, many of which present similar non-GAAP financial measures to investors, and that it allows for greater transparency with respect to key metrics used by management in its financial and operational decision-making.

Management does not consider EBITDA in isolation or as an alternative to financial measures determined in accordance with GAAP. The principal limitation of EBITDA is that it excludes significant expenses that are required by GAAP to be recorded in the company’s financial statements. In order to compensate for these limitations, management presents EBITDA together with GAAP results. Non-GAAP measures should be considered in addition to results prepared in accordance with GAAP, but should not be considered a substitute for, or superior to, GAAP results. A reconciliation table of net loss, the most comparable GAAP financial measure, to EBITDA is included at the end of this release. MaxCyte urges investors to review the reconciliation and not to rely on any single financial measure to evaluate the company’s business.

On May 9, 2022 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that it will host a live conference call and webcast at 8:30 a.m. ET on Monday, May 16, 2022 to report its first quarter 2022 financial results and provide a corporate update (Press release, Syros Pharmaceuticals, MAY 9, 2022, View Source [SID1234613944]).

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To access the live conference call, please dial (866) 595-4538 (domestic) or (636) 812-6496 (international) and refer to conference ID 4664097. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.

On May 9, 2022 Bio-Thera Solutions, Ltd. (SH: 688177), a commercial-stage pharmaceutical company, reported that dosing has begun in a Phase 1 clinical study to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of BAT7104, a bispecific antibody that specifically blocks the interaction of human PD-L1 and CD47 with their corresponding receptors (Press release, BioThera Solutions, MAY 9, 2022, View Source [SID1234613960]).

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BAT7104 was designed to inhibit the PD-1/PD-L1 and CD47/SIRP-α pathways. By an "imbalanced" design with finely tuned binding affinity to CD47 and high affinity to PD-L1, BAT7104 is expected to avoid RBC depletion and block CD47 on CD47+/PD-L1+ tumors in a more selective and tumor-enriched manner. BAT7104 was shown to effectively inhibit both pathways in vitro and demonstrated synergistic activity in inducing complete tumor regression in vivo. In non-human primates (NHP) study, BAT7104 was well tolerated with no adverse effects, suggesting a favorable therapeutic index in future clinical development. "BAT7104 has demonstrated higher anti-tumor activity with better safety profile in preclinical studies". Commented Dr. Shengfeng Li, CEO, Bio-Thera Solutions. "As the first bispecific antibody of Bio-Thera, we are pleased to see BAT7104 enter clinical development in Australia, and we will continue bringing more innovative anti-tumor drugs to cancer patients." Dr. Shengfeng Li continued.

The Phase 1, multi-center, open-label, dose-escalation clinical trial of BAT7104 is designed to assess the safety and tolerability of BAT7104. Key objectives of the study are to determine the maximum tolerated dose and recommended Phase 2 dose (RP2D), and to evaluate pharmacokinetics and preliminary efficacy in patients with advanced solid tumor. Another early stage Phase 1 clinical study evaluating BAT7104 is being conducted in China in patients with solid tumors and lymphoma. In addition, Bio-Thera Solutions is developing several additional innovative oncology assets directed at important IO targets, including PD-1, OX40, CTLA-4, and TIGIT, all in early stage clinical studies.