On November 6, 2019 Emergent BioSolutions Inc. (NYSE: EBS) reported financial results for the third quarter and nine months ended September 30, 2019 (Press release, Emergent BioSolutions, NOV 6, 2019, View Source [SID1234550458]).

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Q3 2019 AND RECENT BUSINESS ACCOMPLISHMENTS

Procurement

Announced the contract award by the Office of the Assistant Secretary for Preparedness and Response (ASPR) in the U.S. Department of Health and Human Services (HHS) valued at approximately $2 billion over 10 years for the continued supply of ACAM2000, (Smallpox (Vaccinia) Vaccine, Live) into the U.S. Strategic National Stockpile (SNS) in support of smallpox preparedness.

Announced the exercise by the Biomedical Advanced Research and Development Authority (BARDA) of the first contract option, valued at $261 million, to procure doses of the next generation anthrax vaccine candidate AV7909 (anthrax vaccine adsorbed with adjuvant) for delivery into the SNS over 12 months.

Awarded a contract by the ASPR in HHS valued at up to $490 million over 10 years ($90 million agreed to now and the remaining $400 million to be negotiated and finalized over the next 6 months) for the continued

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supply of BAT [Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) – (Equine)] into the SNS in support of botulism preparedness and response capability.

Product Development

Granted PRIority MEdicines, or PRIME, designation by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for the company’s chikungunya virus (CHIKV) virus-like particle (VLP) vaccine candidate, CHIKV VLP.

Awarded a research grant by the National Institute on Drug Abuse (NIDA), a component of the National Institutes of Health (NIH), in HHS, valued at approximately $6.3 million over two years, for the continued development of AP007, the company’s sustained-release nalmefene formulation for the treatment of opioid use disorder (OUD).

Awarded approximately $20 million to develop and manufacture an auto-injector containing diazepam (5 mg/mL) to treat nerve agent-induced seizures. Emergent’s device is being designed for intramuscular buddy-administration for use in military environments and for civilian emergencies.

2019 FINANCIAL PERFORMANCE (unaudited)

(I) Quarter Ended September 30, 2019 (Q3)

Revenues

Total Revenues
For Q3 2019, total revenues were $311.8 million, an increase of 80% over 2018. Total revenues reflect the contribution of recently acquired products as well as increased contracts and grants revenue.
Product Sales
For Q3 2019, product sales were $256.2 million, an increase of $122.9 million or 92% as compared to 2018. The increase primarily reflects sales of both NARCAN (naloxone HCl) Nasal Spray, which was acquired in October 2018, and increased sales of ACAM2000, (Smallpox (Vaccinia) Vaccine, Live), offset by decreased sales of Anthrax vaccines (BioThrax (Anthrax Vaccine Adsorbed) and AV7909) and other product sales.
(in millions)
Three Months Ended September 30,

Contract Manufacturing
For Q3 2019, revenue from the Company’s contract manufacturing operations was $20.0 million, a decrease of $2.1 million or 10% as compared to 2018. The decrease primarily reflects contracted service work in Q3 2018 that did not recur in Q3 2019.

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Contracts and Grants
For Q3 2019, revenue from the Company’s development-based contracts and grants was $35.6 million, an increase of $17.4 million as compared to 2018. The increase primarily reflects increased R&D activities related to certain ongoing funded development programs, most notably AV7909.

Operating Expenses

Cost of Product Sales and Contract Manufacturing
For Q3 2019, cost of product sales and contract manufacturing was $108.0 million, an increase of $38.7 million or 56% as compared to 2018. The increase is attributable to the increase in product sales.

Research and Development (Gross and Net)

For Q3 2019, gross R&D expenses were $53.4 million, an increase of $16.4 million or 44% as compared to 2018. The increase primarily reflects costs associated with incremental development programs from the recent acquisitions of PaxVax and Adapt Pharma in October 2018, as well as timing of manufacturing development activities related to the AV7909 program.

For Q3 2019, net R&D expense, which reflects investments made in development programs that are not currently funded in whole or in part by third-party partners and is calculated as gross research and development expenses minus contracts and grants revenue, was $17.8 million, a decrease of $1.0 million or 5% as compared to 2018. The decrease primarily reflects a reduction in raxibacumab technology transfer costs, partially offset by increases related to the development of the CHIKVVLP vaccine and various programs related to opioid overdose response. The Q3 2019 net R&D expense was 6% of adjusted revenue (total revenue less contracts & grants) compared to 12% of adjusted revenue in Q3 2018.

Selling, General and Administrative

For Q3 2019, selling, general and administrative expenses were $65.0 million, an increase of $22.9 million or 54% as compared to 2018. The increase primarily reflects the addition of the operations and integration costs associated with the PaxVax and Adapt Pharma acquisitions.

Amortization of Intangible Assets

For Q3 2019, amortization of intangible assets was $14.7 million versus $3.9 million as compared to 2018. The increase entirely reflects higher non-cash intangible asset amortization costs associated with the PaxVax and Adapt Pharma acquisitions.

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Income Taxes

For Q3 2019, the income taxes expense in the amount of $15.7 million includes the impact of permanent items.

Net Income & Adjusted Net Income

For Q3 2019, the Company recorded net income of $43.2 million, or $0.83 per diluted share, versus net income of $20.9 million, or $0.41 per diluted share, in 2018.

For Q3 2019, the Company recorded adjusted net income of $63.3 million, or $1.21 per diluted share, versus adjusted net income of $28.4 million, or $0.55 per diluted share, in 2018. (1)

EBITDA & Adjusted EBITDA

For Q3 2019, the Company recorded EBITDA of $96.3 million versus $33.7 million in 2018. (1)

For Q3 2019, the Company recorded adjusted EBITDA of $106.4 million versus $39.1 million in 2018. (1)

(II) Nine months ended September 30, 2019 (unaudited)

Revenues

Total Revenues
For the nine months ended September 30, 2019, total revenues were $745.7 million, an increase of 46% over 2018. Total revenues reflect the contribution of recently acquired products as well as increased contracts and grants revenue.

Product Sales
For the nine months ended September 30, 2019, product sales were $592.7 million, an increase of $203.6 million or 52% as compared to 2018. The increase primarily reflects sales of NARCAN Nasal Spray, which was acquired in October 2018, and ACAM2000, offset by decreased sales of Anthrax vaccines (BioThrax and AV7909).
(in millions)
Nine Months Ended September 30,

Contract Manufacturing
For the nine months ended September 30, 2019, revenue from the Company’s contract manufacturing operations was $54.6 million, a decrease of $17.4 million or 24% as compared to 2018. The decrease primarily reflects contracted service work in the nine months of 2018 that did not recur in the nine months of 2019.

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Contracts and Grants
For the nine months ended September 30, 2019, revenue from the Company’s development-based contracts and grants was $98.4 million, an increase of $47.8 million or 94% as compared to 2018. The increase primarily reflects increased R&D activities related to certain ongoing funded development programs, most notably AV7909.

Operating Expenses

Cost of Product Sales and Contract Manufacturing
For the nine months ended September 30, 2019, cost of product sales and contract manufacturing was $300.7 million, an increase of $91.9 million or 44% as compared to 2018. The increase is attributable to the increase in product sales.

Research and Development (Gross and Net)
For the nine months ended September 30, 2019, gross R&D expenses were $163.4 million, an increase of $72.6 million compared to 2018. The increase primarily reflects costs associated with incremental development programs from the recent acquisitions of PaxVax and Adapt Pharma in October 2018, as well as timing of manufacturing development activities related to the AV7909 program.

For the nine months ended September 30, 2019, net R&D expense, which reflects investments made in development programs that are not currently funded in whole or in part by third-party partners and is calculated as gross research and development expenses minus contracts and grants revenue, was $65.0 million, an increase of $24.8 million or 62% as compared to 2018. The increase primarily reflects investments in the development of the CHIKV-VLP vaccine and various programs related to opioid overdose response. The nine months of 2019 net R&D expense was 10% of adjusted revenue (total revenue less contracts & grants) compared to 9% of adjusted revenue in the nine months of 2018.

Selling, General and Administrative
For the nine months ended September 30, 2019, selling, general and administrative expenses were $201.3 million, an increase of $79.5 million or 65% as compared to 2018. The increase primarily reflects the addition of the operations and integration costs associated with the PaxVax and Adapt Pharma acquisitions.

Amortization of Intangible Assets
For the nine months ended September 30, 2019, amortization of intangible assets was $43.9 million versus $11.7 million as compared to 2018. The increase entirely reflects higher non-cash intangible asset amortization costs associated with the PaxVax and Adapt Pharma acquisitions.

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Income Taxes
For the nine months ended September 30, 2019, income tax benefit was $1.7 million, which includes the impact of permanent items.

Net Income & Adjusted Net Income
For the nine months ended September 30, 2019, the Company recorded net income of $7.6 million, or $0.15 per diluted share, versus net income of $66.2 million, or $1.29 per diluted share, in 2018.

For the nine months ended September 30, 2019, the Company recorded adjusted net income of $67.0 million, or $1.28 per diluted share, versus adjusted net income of $83.3 million, or $1.63 per diluted share, in 2018. (1)

EBITDA & Adjusted EBITDA
For the nine months ended September 30, 2019, the Company recorded EBITDA of $116.3 million versus $115.5 million in 2018. (1)

For the nine months ended September 30, 2019, the Company recorded adjusted EBITDA of $145.4 million versus $124.6 million in 2018. (1)

2019 FINANCIAL FORECAST (Reaffirmed)

* Reflects revision from previous guidance ($255-$285) due to the inclusion of changes in fair value of contingent consideration as an additional item for reconciliation in the calculation of Adjusted EBITDA; management has determined Adjusted EBITDA is more reflective of the Company’s true operations than EBITDA alone and the guidance range for EBITDA has been revised based on current expectations.

The Company’s financial forecast for 2019 includes the anticipated impact of full year product sales, continued contract manufacturing and contracts & grants revenue as well as continued investment in internally funded development projects. The outlook for 2019 does not include estimates for potential new corporate development or other M&A transactions.

FOOTNOTES

(1) See "Reconciliation of Net Income to Adjusted Net Income, EBITDA and Adjusted EBITDA" for a definition of terms and a reconciliation table.

CONFERENCE CALL AND WEBCAST INFORMATION
Company management will host a conference call at 5:00 pm (Eastern Time) today, November 6, 2019, to discuss these financial results. This conference call can be accessed live by telephone or through the Company’s website.

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Live Teleconference Information:
Dial in: [US] (855) 766-6521; [International] (262) 912-6157
Conference ID: 1169597

Live Webcast Information:
Visit View Source for the live webcast.

On November 6, 2019 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing novel small molecule therapeutics to treat unmet needs in autoimmunity and cancer, reported its third quarter 2019 financial results and corporate highlights (Press release, Kezar Life Sciences, NOV 6, 2019, View Source [SID1234550475]).

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"I commend our team’s continued execution across our clinical and preclinical programs here at Kezar. Our three Phase 2 trials with KZR-616 in severe autoimmune diseases are progressing on track, and we look forward to disclosing additional data from our MISSION study at the ACR meeting next week", said John Fowler, Chief Executive Officer. "Additionally, the nomination of the first clinical candidate from our protein secretion program is a pivotal step for the company and underscores the depth and breadth of our R&D capabilities. Our small molecule approach to targeting the Sec61 translocon represents a highly compelling new therapeutic approach, and the preclinical data generated with KZR-261 gives us confidence that it may prove effective in treating a variety of tumor types. We are excited to share more about this highly novel target and illustrate the broad platform potential of this approach."

Recent Clinical and Business Highlights

KZR-616 – Selective Immunoproteasome Inhibitor

MISSION Study

The Phase 1b/2 MISSION study in systemic lupus erythematosus (SLE) patients with and without nephritis is currently ongoing.

On October 1, 2019, we presented a corporate and strategic update, including a protocol amendment for the Phase 2 portion of the MISSION study (NCT03393013). The updated protocol is designed to generate more robust and meaningful data around the safety and efficacy of KZR-616 in lupus nephritis, including the evaluation of three dose levels of KZR-616 (administered with background medication) for a treatment period of six months.

The Phase 1b portion of the MISSION study is ongoing and additional data will be presented during a poster session at the 2019 American College of Rheumatology (ACR) Meeting in Atlanta, GA on November 12, 2019.

PRESIDIO Study

In August 2019, we announced the initiation of the PRESIDIO study (NCT04033926), a Phase 2 randomized, double-blind, placebo-controlled, crossover, multicenter study to evaluate the safety, tolerability, efficacy, PK and PD of treatment with KZR-616 in patients with active dermatomyositis (DM) or polymyositis (PM). This trial is expected to enroll 24 patients with either DM or PM.

MARINA Study

In August 2019, we announced the initiation of the MARINA study (NCT04039477), a Phase 2 randomized, dose-blind, multicenter study to evaluate the safety and efficacy of KZR-616 in the treatment of patients with autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP). This trial is expected to enroll 40 patients with either AIHA or ITP.

KZR-261 – Protein Secretion Program

Our research and discovery efforts targeting the protein secretion pathway have progressed significantly, and today, we announce the nomination of KZR-261 as our first oncology clinical candidate. KZR-261 has demonstrated broad anti-tumor activity in preclinical models of both solid and hematologic malignancies, and we have initiated laboratory studies and manufacturing activities towards an Investigational New Drug (IND) filing for a Phase 1 study in solid tumors, which we anticipate occurring in Q1 2021.

Additionally, this novel program and pathway will be featured in four separate presentations during two major medical and scientific conferences: the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) and the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting & Exposition (ASH) (Free ASH Whitepaper). The abstract titles are summarized below.

SITC: November 6-10, 2019, National Harbor, MD

Abstract Number: 815

Title: Targeting multiple immune checkpoint proteins with novel small molecule inhibitors of Sec61-dependent cotranslational translocation

Date: Friday, November 8, 2019

Time: 12:30pm – 2:00pm

Poster Session: Novel Single-Agent Immunotherapies

ASH: December 7-10, 2019, Orlando, FL

Oral Presentations

Abstract Number: 408

Title: Blocking Protein Secretion with Novel Small Molecule Inhibitors of Sec61 Represents a Potential Treatment Strategy Against Hematologic Malignancies

Date: Sunday, December 8, 2019
Time: 10:45am

Oral Session: 802. Chemical Biology and Experimental Therapeutics: Novel Compounds and Mechanisms of Action

Abstract Number: 805
Title: Protein Translocation Inhibitors Overcome Cytokine-Induced Glucocorticoid Resistance in T-Cell Acute Lymphoblastic Leukemia

Date: Monday, December 9, 2019
Time: 4:30 PM

Oral Session: 605. Molecular Pharmacology, Drug Resistance—Lymphoid and Other

Poster Presentation

Abstract Number: 2076

Title: Proteomic Profiling and Mechanistic Investigating of a Novel Anti-Cancer Small Molecule Inhibitor of Sec61
Date: Saturday, December 7, 2019
Time: 5:30 PM – 7:30 PM

Poster Session: 802. Chemical Biology and Experimental Therapeutics: Poster I

Financial Results

Cash, cash equivalents and marketable securities totaled $85.2 million as of September 30, 2019, compared to $107.4 million as of December 31, 2018. The decrease in cash, cash equivalents and marketable securities was primarily attributable to cash used by the company in operations to advance its clinical stage programs as well as preclinical research and development.

Research and development expenses for the third quarter of 2019 increased by $2.4 million to $7.1 million compared to $4.7 million in the third quarter of 2018. This increase was primarily related to advancing both the KZR-616 clinical program in multiple indications and the protein secretion preclinical program.

General and administrative expenses for the third quarter of 2019 increased by $1.0 million to $2.6 million compared to $1.6 million in the third quarter of 2018. The increase was primarily due to an increase in personnel expenses and costs related to operating as a public company.

Net loss for the third quarter of 2019 was $9.1 million, or $0.48 per basic and diluted common share, compared to a net loss of $5.7 million, or $0.30 per basic and diluted common share, for the third quarter of 2018.

Total shares outstanding were 19.1 million as of September 30, 2019. Additionally, there were outstanding options to purchase 3.3 million shares of common stock at a weighted average exercise price of $7.39 per share as of September 30, 2019.

About KZR-616

KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Phase 1a clinical trial results in healthy volunteers provide evidence that KZR-616 potentially avoids adverse effects caused by currently

marketed non-selective proteasome inhibitors, which we believe prevent them from being utilized as a chronic treatment in autoimmune disorders. Phase 2 trials are underway for the treatment of lupus nephritis (MISSION study), dermatomyositis and polymyositis (PRESIDIO study), and autoimmune hemolytic anemia and immune thrombocytopenia (MARINA study).

About KZR-261

KZR-261, a novel, first-in-class protein secretion inhibitor, is the first clinical candidate to be nominated from our research and discovery efforts targeting protein secretion pathways as potential therapies for oncology, immuno-oncology and autoimmune indications. KZR-261 is a broad-spectrum anti-tumor agent that acts through direct interaction and inhibition of Sec61 activity. The compound was discovered at Kezar through a medicinal chemistry campaign in which several scaffolds were progressed through the company’s proprietary work flow of Sec61 modulation. As a result, Kezar has established a unique and broad library of protein secretion inhibitors and a strong patent position around KZR-261 and its analogs. KZR-261 has demonstrated several encouraging features that lend to its potential to be a new anti-cancer agent for the treatment of solid and hematologic malignancies. IND-enabling studies are currently underway, and an IND filing in solid tumors is expected in Q1 2021.

On November 6, 2019 AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX), (AcelRx), a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for use in medically supervised settings, reported its third quarter 2019 financial results (Press release, AcelRx Pharmaceuticals, NOV 6, 2019, View Source [SID1234550517]).

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"Achieving 125 REMS-certified facilities two months ahead of our year-end goal demonstrates healthcare providers’ increasing interest in DSUVIA," said Vince Angotti, Chief Executive Officer of AcelRx. "We also expect to achieve our year-end objective of 125 formulary approvals and anticipate expanded use across a variety of healthcare settings as the feedback on DSUVIA from healthcare professionals continues to be very positive," continued Angotti.

Third Quarter and Recent Highlights

130 healthcare facilities are now REMS-certified and able to purchase DSUVIA and 105 formulary approvals have been achieved through October 31, 2019; approximately 60% of REMS certifications and formulary approvals have occurred since the date of the Company’s last earnings call
AcelRx was selected to present at one of the two plenary sessions of the 2019 Military Health System Research Symposium; the presentation was entitled "Pooled Safety Analysis of Patients Who Were Exposed to < 300 mcg vs. ≥300 mcg of Sublingual Sufentanil in a 24 Hour Period for Treatment of Acute Pain"
Pooled dosing and efficacy data from use of the sufentanil sublingual tablet (SST) 30 mcg among multiple demographic subgroups (age, sex, race, and body mass index) was accepted for publication in the Journal of PeriAnesthesia Nursing to aid nurses in understanding the effectiveness and duration of action of SST 30 mcg in the management of moderate-to-severe acute pain across a variety of patient demographics
Financial Information

Cash, cash equivalents and short-term investments balance of $80.4 million as of September 30, 2019;
Combined R&D and SG&A expenses for the third quarter of 2019 totaled $12.0 million compared to $8.8 million for the third quarter of 2018. Excluding stock-based compensation expense, these amounts were $10.7 million for the third quarter of 2019 compared to $7.1 million for the third quarter of 2018. R&D and SG&A expenses for the first nine months of 2019 totaled $35.8 million compared to $23.6 million in the first nine months of 2018. Excluding stock-based compensation expense, these figures were $32.3 million for the first nine months of 2019 compared to $19.9 million for the first nine months of 2018. The increase in R&D and SG&A expenses is primarily due to increased personnel-related expenses for the commercial launch of DSUVIA. See the "Reconciliation of Non-GAAP Financial Measures" table below for a reconciliation of the non-GAAP operating expenses described above to their related GAAP measures;
Net cash outflow for the third quarter of 2019 was $11.1 million, which included $0.6 million in debt service;
For the third quarter of 2019, net loss was $12.7 million, or $0.16 per basic and diluted share, compared to $12.5 million, or $0.21 per basic and diluted share, for the third quarter of 2018. Net loss for the first nine months of 2019 was $38.8 million, or $0.49 per basic and diluted share, compared to $34.6 million, or $0.64 per basic and diluted share, for the prior year period.
Webcast and Conference Call Information
As previously announced, AcelRx will host a live webcast Wednesday, November 6, 2019 at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time) to discuss these financial results and provide other corporate updates. The webcast is accessible by visiting the Investors page of the Company’s website at www.acelrx.com and clicking on the webcast link. The webcast will be accompanied by a slide presentation. Investors who wish to participate in the conference call may do so by dialing (866) 361-2335 for domestic callers, (855) 669-9657 for Canadian callers or (412) 902-4204 for international callers. A webcast replay will be available on the AcelRx website for 90 days following the call by visiting the Investor page of the Company’s website at www.acelrx.com.

About DSUVIA (sufentanil sublingual tablet), 30 mcg
DSUVIA, known as DZUVEO in Europe, approved by the FDA in November 2018, is indicated for use in adults in certified medically supervised healthcare settings, such as hospitals, surgical centers, and emergency departments, for the management of acute pain severe enough to require an opioid analgesic, and for which alternative treatments are inadequate. DSUVIA was designed to provide rapid analgesia via a non-invasive route and to eliminate dosing errors associated with intravenous (IV) administration. DSUVIA is a single-strength solid dosage form administered sublingually via a single-dose applicator (SDA) by healthcare professionals. Sufentanil is an opioid analgesic previously only marketed for IV and epidural anesthesia and analgesia. The sufentanil pharmacokinetic profile when delivered sublingually avoids the high peak plasma levels and short duration of action observed with IV administration. The European Commission approved DZUVEO for marketing in Europe in June 2018 and the Company is currently in discussions with potential European marketing partners.

On November 6, 2019 MaaT Pharma reported that a poster will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting with initial data on it’s lead microbiome restoration biotherapeutic, MaaT013, as a treatment in patients with gastrointestinal-predominant, Steroid-Refractory and Steroid-Dependent acute Graft-versus-Host-Disease (GI aGvHD) following allogeneic Hematopoietic Stem-Cell Transplantation (allo-HSCT) (Press release, MaaT Pharma, NOV 6, 2019, View Source [SID1234550531]). MaaT Pharma provided the product as a pharmaceutical preparation to hospitals as part of its compassionate use program for patients who had previously received and failed up to five previous systemic treatments for GvHD. MaaT013 is an enema formulation of a microbiota biotherapeutic characterized by a high diversity and consistent richness of microbial species derived from pooled healthy donors. The product is manufactured at the company’s centralized European cGMP production facility using its proprietary microbiome restoration biotherapeutic platform. The poster will be presented during the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando, Florida from December 7 – 10, 2019. The poster abstract was published in the online meeting program on November 6, 2019 at 9:00 AM EDT and also in the online November supplemental issue of the journal Blood.

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Poster Presentation details:

Title: Successful and Safe Treatment of Intestinal Graft-Versus-Host Disease (GvHD) with Pooled-Donor Full Ecosystem Microbiota Biotherapeutics
Abstract No: 1993
Session Name: 722. Clinical Allogeneic Transplantation: Acute and Chronic GvHD, Immune Reconstitution: Poster I
Date/Time: Saturday, December 7, 2019 / 5:30 PM – 7:30 PM EDT
Location: Orange County Convention Center, Hall B

MaaT Pharma will announce the results through a press release following the presentation and will further make the poster available on the company website under "News".

In addition to the compassionate use program, MaaT Pharma is currently investigating MaaT013 in a Phase II clinical trial in acute SR GI GvHD patients (NCT03359980). The study, named HERACLES, is a multi-center, single-arm, open-label study, enrolling 32 patients to evaluate the safety and efficacy of MaaT013 in steroid-resistant, gastrointestinal-predominant aGvHD (SR GI aGvHD) patients. Acute GvHD is a serious, often fatal syndrome typically involving the gut, skin and liver. MaaT Pharma has established the most complete approach to restoring patient-microbiome symbiosis in the gut to improve efficacy of cancer treatments and survival outcomes in life-threatening diseases. The company announced in June this year the completion of a third positive safety assessment of MaaT013 in the Phase II trial by an independent Data and Safety Monitoring Board (DSMB). MaaT013 has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

On November 6, 2019 Sierra Oncology, Inc. (Nasdaq: SRRA), a late-stage drug development company focused on the development and commercialization of momelotinib, a JAK1, JAK2 & ACVR1 inhibitor with a potentially differentiated therapeutic profile for the treatment of myelofibrosis, reported that new analyses of RBC transfusion data from SIMPLIFY-1, a double-blind Phase 3 trial of its investigational drug momelotinib vs ruxolitinib in JAK inhibitor naïve patients, will be presented in a poster at the 61st American Society of Hematologists (ASH) (Free ASH Whitepaper) Annual Meeting in Orlando, Florida (Press release, Sierra Oncology, NOV 6, 2019, View Source [SID1234550547]). These retrospective analyses, to be presented by renowned myelofibrosis expert, Dr. Ruben Mesa, demonstrate that patients who received momelotinib had significantly decreased transfusion requirements compared to those treated with ruxolitinib, including a decreased risk of receiving a transfusion and a longer transfusion-free period.

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"We explored the relative burden of transfusions, a tangible and significant concern to clinicians and patients with myelofibrosis, by examining the SIMPLIFY-1 data comprising more than 400 myelofibrosis patients through statistical models utilizing a variety of novel dynamic anemia benefit endpoints, including the proportion of patients who remained transfusion free, the odds of requiring zero transfusions, the time to first transfusion in patients who remain transfusion requiring and the overall transfusion burden," said Dr. Ruben Mesa, Director of the Mays Cancer Center, home to UT Health San Antonio MD Anderson Cancer Center. "Overall, these highly persuasive statistical analyses further confirm that momelotinib treatment elicits a substantive mechanistically-driven anemia benefit, including a significantly greater proportion of momelotinib patients remaining transfusion free, a 50% lower risk of receiving a transfusion and nearly 10-fold higher odds of receiving no transfusions during the 24-week study period, directly compared to ruxolitinib."

Historically, myelofibrosis clinical studies have employed landmark analyses of transfusion independence or dependence to describe anemia benefit. While momelotinib is the only JAK inhibitor to have demonstrated superior head-to-head landmark anemia benefits compared to ruxolitinib, these static measures of anemia alone do not fully describe the significant, clinically meaningful anemia benefit observed following momelotinib administration. In conjunction with the statistical consulting group at the International Drug Development Institute (IDDI), Sierra has developed additional sophisticated dynamic statistical analyses of anemia benefit in order to complement those assessments typically presented in the myelofibrosis literature.

"Chronic, progressive anemia is a key hallmark feature of myelofibrosis and the most important negative prognostic indicator of reduced survival in this disease. Approximately 60% of patients are anemic and 45% are transfusion dependent within one year of diagnosis, with most patients ultimately progressing to transfusion dependency. Unfortunately, currently approved JAK inhibitor therapies induce or worsen anemia, exacerbating this significant unmet medical need in anemic myelofibrosis patients," said Dr. Barbara Klencke, Chief Development Officer of Sierra Oncology. "The marked systemic inflammation seen in myelofibrosis leads to increased ACVR1 activity which in turn increases secretion of hepcidin, resulting in perturbed iron homeostasis and an iron-restricted anemia. Momelotinib’s inhibition of ACVR1 in addition to JAK1 and JAK2, unique amongst the JAK inhibitor class, results in notable reductions of both hepcidin and inflammation, restoring iron homeostasis and RBC production, thereby alleviating anemia and transfusion dependency."

The SIMPLIFY-1 trial is a double-blind, active-controlled Phase 3 study in which 432 patients received randomized treatment with momelotinib or ruxolitinib for 24 weeks (JCO. 2017;35:3844–50). In addition to a significant reduction in splenomegaly and improvements in constitutional symptoms, previously reported analyses of the SIMPLIFY-1 data demonstrated that patients in the momelotinib arm achieved nominal-statistical significance for all anemia endpoints tested, including a higher rate of transfusion independence (p < 0.001) and lower rates of transfusion dependence (p = 0.019) at Week 24, compared to patients on ruxolitinib, consistent with momelotinib’s pro-erythropoietic effect.

New retrospective analyses of the SIMPLIFY-1 transfusion data demonstrated that:

The Kaplan-Meier (K-M) proportion of patients who did not require any transfusions during the randomized treatment period was significantly greater for the momelotinib treated group (73%) than those who received ruxolitinib (46%; p<0.0001)
The K-M proportion of patients requiring fewer than 5 RBC units over the treatment period was also significantly greater for momelotinib (83%) than ruxolitinib (62%; p<0.0001).
The odds of a momelotinib patient receiving no transfusions was approximately 10-fold higher including covariates, compared to a patient treated with ruxolitinib
The overall transfusion burden during momelotinib therapy was approximately half of that for ruxolitinib (HR 0.522; p<0.0001) for models both with and without baseline characteristics from patients as covariates.
These dynamic measures of transfusion requirement will be further evaluated as exploratory endpoints in MOMENTUM, a planned Phase 3 study of momelotinib in anemic myelofibrosis patients previously treated with a JAK inhibitor, anticipated to launch in the fourth quarter of 2019. The MOMENTUM trial has been designed to re-confirm momelotinib’s ability to address each of the three hallmark features of myelofibrosis, namely constitutional symptoms, anemia, and splenomegaly. Dr. Srdan Verstovsek, MD, PhD, Chief, Section for Myeloproliferative Neoplasms, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas, has been named Chief Investigator of the trial.

About the ASH (Free ASH Whitepaper) 2019 Poster:
Title: Dynamic and Time-To-Event Analyses Demonstrate Marked Reduction in Transfusion Requirements for Janus Kinase Inhibitor–Naïve Myelofibrosis Patients Treated with Momelotinib Compared Head to Head with Ruxolitinib
Session Name: 634. Myeloproliferative Syndromes: Clinical: Poster I
Date: Saturday, December 7, 2019
Presentation Time: 5:30 PM – 7:30 PM
Location: Orange County Convention Center, Hall B