On August 9, 2021 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported the receipt of notification from its partner, AbbVie Inc., regarding its termination of the license and collaboration agreement for the investigational drug abicipar pegol for the treatment of neovascular age-related macular degeneration (nAMD) and Diabetic Macular Edema (DME) (Press release, Molecular Partners, AUG 9, 2021, View Source [SID1234586136]). As such, Molecular Partners will regain the development and commercial rights of abicipar on a worldwide basis.

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"There remains a significant unmet medical need for patients living with nAMD and DME, and we remain confident in abicipar’s potential to offer these patients a differentiated treatment option over existing therapies," said Patrick Amstutz, Chief Executive Officer of Molecular Partners. "Our focus for this program will be determining the best path to value creation within the context of our expansive portfolio of antiviral and immuno-oncology therapies in development."

Molecular Partners will form a special committee to evaluate the program and determine appropriate next steps. In addition, Molecular Partners and AbbVie will continue their ongoing discovery alliance, in which AbbVie will continue to evaluate additional DARPin candidates for ophthalmic indications. The return of the abicipar program is not expected to impact Molecular Partners’ financial outlook for 2021 or previously issued guidance.

Abicipar is a long-acting anti-VEGF DARPin molecule which was invented by Molecular Partners and initially licensed to Allergan in 2011. The program has been through two positive Phase 3 studies, CEDAR and SEQUOIA, which supported the non-inferior efficacy of the abicipar quarterly dosing regimen to maintain vision gains with more than 50 percent fewer injections versus ranibizumab (13 vs. 6) dosed monthly in the first year.

With the acquisition of Allergan by AbbVie, the rights to abicipar were transferred to AbbVie. In June 2020, AbbVie received a Complete Response Letter to the Biologics License Application for abicipar pegol, indicating that the rate of intraocular inflammation observed following administration of Abicipar pegol resulted in an unfavorable benefit-risk ratio in the treatment of nAMD (AMD), and that additional work would be required to demonstrate a lower rate of ocular inflammation than what was previously seen in the Phase 3 studies.

On August 9, 2021 Sierra Oncology, Inc. (NASDAQ: SRRA), a late-stage biopharmaceutical company on a mission to deliver targeted therapies that treat rare forms of cancer, reported guidance on the development timeline for its lead product candidate, momelotinib (Press release, Sierra Oncology, AUG 9, 2021, View Source [SID1234586153]). The company now expects topline data from the Phase 3 registration-enabling MOMENTUM study by February 2022, and assuming positive results, the company plans to file a New Drug Application with the U.S. Food & Drug Administration in Q2 2022.

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Sierra also announced it has raised $34 million in gross proceeds thus far in Q3 2021 through the use of its at-the-market (ATM) offering. These funds are in addition to the $90.7 million in cash and cash equivalents on hand at the close of the second quarter of 2021, further bolstering the company’s cash balance as it prepares for momelotinib commercialization. The company also has the potential to obtain up to $33.3 million in cash from Series B warrants that must be exercised within 75 days of the announcement of MOMENTUM topline data.

Stephen Dilly, MBBS, PhD, President and Chief Executive Officer of Sierra Oncology noted, "The continued excellent execution of the MOMENTUM study and further acceleration of the timeline, combined with the additional funding secured over the last few weeks, puts us in a very strong position as we approach topline data."

On August 9, 2021 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 second quarter ended June 30, 2021. In addition, the Company provided a clinical and business update (Press release, Syndax, AUG 9, 2021, View Source [SID1234586105]).

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"The second quarter of 2021 was marked by significant progress advancing our pipeline of innovative therapeutics, and we are pleased to share that we have selected a go-forward dose for the pivotal Phase 2 portion of AUGMENT-101 based on favorable findings from the intermediate dose cohort," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "Enrollment in the trial continues, with recently enrolled patients expected to count towards the Phase 2 expansion portion, subject to endorsement of the Phase 2 dose by the U.S. Food and Drug Administration (FDA). We are also excited to announce an important step in our plans to expand development of SNDX-5613 into earlier lines of therapy with the initiation of the first frontline combination trial of a menin inhibitor in the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial."

"Beyond SNDX-5613, enrollment remains on track in the ongoing global pivotal AGAVE-201 trial of axatilimab in patients with chronic graft versus host disease (cGVHD), and we continue to expect topline data from the trial in 2023. In addition, as previously announced, we anticipate sharing updated results from the recently completed Phase 1/2 trial of axatilimab in cGVHD later this year and remain committed to unlocking the full potential of axatilimab in the growing number of cGVHD patients lacking effective interventions."

Recent Progress and Anticipated Milestones

SNDX-5613

In May 2021, the Company reported updated data from the ongoing Phase 1 dose escalation portion of the Phase 1/2 AUGMENT-101 trial of SNDX-5613, a highly selective oral menin inhibitor, in patients with mixed lineage leukemia rearranged (MLLr) and nucleophosmin (NPM1c) mutant relapsed/refractory (R/R) acute leukemias. The updated May data showed that a total of 7/31 patients (23%) have achieved CR/CRh.

Based on positive findings from the intermediate dose cohort, in which no dose limiting toxicities were observed, the Company has selected 276 mg of SNDX-5613 every 12 hours in patients who are not receiving a concomitant strong CYP3A4 inhibitor (Arm A) and 163 mg every 12 hours for patients who are receiving a concomitant strong CYP3A4 inhibitor (Arm B), for Phase 2. Enrollment in the trial remains ongoing, with recently enrolled patients expected to count towards the Phase 2 expansion portion, subject to endorsement of the Phase 2 dose by the U.S. FDA.

The Company reported it will initiate a frontline trial of SNDX-5613 in combination with venetoclax and azacytidine in newly diagnosed acute myeloid leukemia (AML) patients unable to tolerate induction chemotherapy. The trial will be conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial, a collaborative clinical trial that aims to change the paradigm of AML treatment through a precision medicine approach. SNDX-5613 is the first menin inhibitor to be included in the Beat AML Master Clinical Trial.

The Company also announced today that it plans to initiate a new trial to assess the safety, tolerability, and preliminary anti-leukemic efficacy of SNDX-5613 in combination with chemotherapy in patients with R/R MLLr or NPM1 acute leukemias. The Phase 1b trial, which will be referred to as AUGMENT-102, is expected to enroll up to 27 patients.

In June 2021, the Company announced that the U.S. FDA granted Fast Track Designation (FTD) to SNDX-5613 for the treatment of adult and pediatric patients with R/R acute leukemias harboring an MLLr or NPM1 mutation. FTD is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier.
Axatilimab

In May 2021, Syndax announced that enrollment is complete in the Phase 2 expansion portion of the Phase 1/2 trial of axatilimab, its anti-CSF-1R monoclonal antibody, in patients with cGVHD. The Company continues to anticipate reporting updated results at a medical meeting in the fourth quarter of 2021 for 40 patients, including the 17 patients in the Phase 1 portion and 23 patients from the Phase 2 expansion portion, which evaluated 1 mg/kg of axatilimab every two weeks. At the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2020, preliminary data from the Phase 1 portion of the trial were reported during an oral presentation highlighting the tolerability and high response rate of axatilimab in cGVHD patients refractory to multiple therapeutic agents.

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 2023. The trial will evaluate 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.

Earlier this year, the Company announced that the U.S. FDA granted Orphan Drug Designation to axatilimab for the treatment of patients with cGVHD and idiopathic pulmonary fibrosis.
Second Quarter 2021 Financial Results

As of June 30, 2021, Syndax had cash, cash equivalents and short-term investments of $253.1 million and 51.9 million shares and share equivalents issued and outstanding. This includes 3.3 million pre-funded warrants.

Second quarter 2021 research and development expenses increased to $16.9 million from $10.9 million for the prior year period. The increase was primarily due to increased clinical trial activities and increased CMC activities.

General and administrative expenses for the second quarter 2021 decreased to $5.8 million from $6.0 million for the prior year period. The decrease is primarily due to the absence of pre-commercialization expenses for entinostat in 2021 partially offset by employee related expenses.

For the three months ended June 30, 2021, Syndax reported a net loss attributable to common stockholders of $22.9 million or $0.44 per share compared to $17.1 million or $0.42 per share for the prior year period.

Financial Update and Guidance

For the third quarter of 2021, research and development expenses are expected to be $25 to $30 million, and total operating expenses are expected to be $30 to $35 million. For the full year of 2021, research and development expenses are expected to be $90 to $100 million, and total operating expenses are expected to be $110 to $120 million.

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, Monday, August 9, 2021.

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

On August 9, 2021 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies, reported its financial results for the second quarter ended June 30, 2021 and provided an update on recent developments (Press release, IGM Biosciences, AUG 9, 2021, View Source [SID1234586121]).

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"We continue to make steady progress in the development of our innovative product pipeline, including the successful completion of the initial dose escalation portion of our IGM-2323 Phase 1 clinical trial," said Fred Schwarzer, Chief Executive Officer of IGM Biosciences. "We look forward to announcing a recommended Phase 2 dose for IGM-2323, presenting initial dose escalation data from the Phase 1 trial of IGM-8444, initiating a Phase 1 trial for IGM-6268 and filing an IND for IGM-7354 this year. "

Pipeline Updates

IGM-2323

Phase 1 dose escalation completed; dose expansion continues. IGM continues to advance the clinical development of IGM-2323, the Company’s IgM-based CD20 x CD3 bispecific antibody, for the treatment of non-Hodgkin’s lymphoma (NHL). The Company cleared its highest planned dose escalation cohort, a top dose of 1000 mg, without a dose limiting toxicity, and is currently treating additional patients in four Phase 1 dose cohorts with top doses of 100 mg, 300 mg, 600 mg and 1000 mg, respectively. IGM expects to select a recommended Phase 2 dose in 2021.
IGM-8444

Additional dose cohorts cleared in Phase 1. IGM continues to advance the clinical development of IGM-8444, the Company’s IgM Death Receptor 5 (DR5) agonist, for the treatment of a potentially broad range of solid tumors and hematologic malignancies. IGM has cleared its third dose cohort (3 mg/kg) of the single-agent portion of its Phase 1 clinical study and is currently treating patients in its highest dose escalation cohort (10 mg/kg) with every two-week single agent dosing. IGM has also cleared the first dose cohort of the FOLFIRI combination portion of the Phase 1 study and is currently treating patients in the second of four planned FOLFIRI combination dose escalation cohorts. IGM expects to report initial data from the dose escalation portion of the Phase 1 trial in 2021.
Clinical testing of birinapant in combination with IGM-8444 expected to begin this year. IGM remains on track to begin clinical testing of birinapant in combination with IGM-8444 in 2021.
IGM-6268

New pipeline candidate for the treatment and prevention of COVID-19 expected to advance into the clinic this year. In June 2021, IGM announced IGM-6268, which represents the expansion of the Company’s IgM platform into infectious diseases. IGM-6268 is an IgM version of an anti-SARS-CoV-2 IgG monoclonal antibody and is being developed as an intranasally administered agent for the treatment and prevention of COVID-19. IGM expects to initiate a Phase 1 clinical trial of IGM-6268 in 2021.
Nature manuscript published. In June 2021, Nature published an article entitled "Nasal delivery of an IgM offers broad protection from SARS-CoV-2 variants". The article describes results from preclinical studies demonstrating significantly greater neutralization of SARS-CoV-2 with an IgM antibody compared to IgG antibodies, the potent neutralization of all evaluated mutant Variants of Concern and Variants of Interest, and the ability to provide effective preventative and therapeutic protection when delivered intranasally in mice. The article was co-authored by researchers at IGM, The University of Texas Medical Branch at Galveston and The University of Texas Health Science Center at Houston.
IGM-7354

Investigational New Drug (IND) application expected to be filed this year. IGM plans to file an IND application with the U.S. Food and Drug Administration (FDA) for IGM-7354, the Company’s IL‑15 x PD‑L1 bispecific IgM antibody, before the end of 2021 in order to begin clinical testing initially in solid tumors, followed by hematologic malignancies.
Corporate Updates

Chris Takimoto appointed Chief Medical Officer. Dr. Takimoto brings 30 years of experience in cancer research and development. Most recently, Dr. Takimoto was Senior Vice President, Oncology at Gilead Sciences, Inc. Prior to Gilead, he served as Chief Medical Officer of Forty Seven, Inc., a biotechnology company formed out of Stanford University and acquired by Gilead Sciences in 2020.
Second Quarter 2021 Financial Results

Cash and Investments: Cash and investments as of June 30, 2021 were $301.8 million, compared to $366.3 million as of December 31, 2020.
Research and Development (R&D) Expenses: For the second quarter of 2021, R&D expenses were $30.1 million, compared to $15.0 million for the same period in 2020.
General and Administrative (G&A) Expenses: For the second quarter of 2021, G&A expenses were $8.6 million, compared to $4.4 million for the same period in 2020.
Net Loss: For the second quarter of 2021, net loss was $38.7 million, or a loss of $1.16 per share, compared to a net loss of $18.8 million, or a loss of $0.62 per share, for the same period in 2020.
2021 Financial Guidance

IGM reiterates its previously issued financial guidance expecting full year GAAP operating expenses to be between $175 million and $185 million including estimated non-cash stock-based compensation expense of approximately $25 million. IGM expects to end 2021 with a balance of over $200 million in cash and investments.

On August 9, 2021 Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) ("Eagle" or the "Company") reported that it has entered into a licensing agreement with AOP Orphan Pharmaceuticals GmbH ("AOP Orphan"), a privately owned Austrian company devoted to the treatment of rare and special diseases, for the commercial rights to its product, Landiolol in the United States (Press release, Eagle Pharmaceuticals, AUG 9, 2021, View Source [SID1234586137]). Landiolol, a leading hospital emergency use product, is currently approved in Europe for the treatment of non-compensatory sinus tachycardia and tachycardic supraventricular arrhythmias. The Company will support the submission of a new drug application ("NDA") to the U.S. Food and Drug Administration ("FDA") seeking approval for Landiolol for the short-term reduction of ventricular rate in patients with supraventricular tachycardia ("SVT"), including atrial fibrillation and atrial flutter.

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Landiolol is a short-acting, ultra-high selective beta-1 adrenoceptor blocker developed by AOP Orphan that has a selective effect on heart rate over cardiac contractility. Landiolol is available in two forms (20 mg/2ml concentrate, 300 mg powder) and is designed for use in emergency, cardiac critical care, operating room, and intensive care settings. It is registered in several European countries for the treatment of non-compensatory sinus tachycardia and tachycardic supraventricular arrhythmias. The drug uses a proprietary dosing algorithm to facilitate the administration.

Under the terms of the agreement, Eagle will facilitate the U.S. regulatory pathway for the approval of Landiolol. In addition, Eagle will be responsible for the U.S. commercialization of the product upon approval. Landiolol, which has not previously been marketed in the U.S., is covered by several patents, and the Company anticipates five years of new chemical entity ("NCE") exclusivity.

Landiolol is already commercially available in Japan (Onoact) and several European markets as RAPIBLOC. A review of multiple clinical studies suggests that Landiolol is a useful option for the rapid short-term control of tachyarrhythmias (Syed YY. Landiolol: A Review in Tachyarrhythmias. Drugs. 2018 Mar;78(3):377-388. doi: 10.1007/s40265-018-0883-9. PMID: 29470800.). A Type C meeting was held with FDA in July 2020, at which time AOP Orphan proposed a submission strategy in which it would provide summaries of pre-existing safety and efficacy data and a meta-analysis of published randomized controlled trials. FDA tentatively agreed with this methodological approach and deemed data sets adequate to support a proposed NDA.

"This is an exciting near-term opportunity for Eagle, with the potential to file an NDA in the first quarter of next year. The clinical advantages of Landiolol are well recognized within the medical community, and we look forward to advancing this asset for FDA approval in the United States. Our deep understanding of the U.S. regulatory landscape, along with our established research and development infrastructure, will be valuable in accelerating the program. Once approved, we plan to leverage our current sales force and relationships in the critical care setting to promote the product. There is broad potential to expand the portfolio of future indications for Landiolol’s use," stated Scott Tarriff, Chief Executive Officer of Eagle Pharmaceuticals.

"With this license agreement, we are solidifying our hospital and critical care product portfolio, as we look to capitalize on multiple near- and longer-term opportunities. As we have stated, executing on our growth strategy for Eagle beyond 2021 has been a priority. With the anticipated launch of vasopressin, the February 2022 launch of PEMFEXY, the recent launch of bendamustine in Japan, our current pipeline, and now the future potential Landiolol launch, we believe we have a firm foundation for sustained future growth," concluded Tarriff.

"The step into the American market forms the basis for further expansion of AOP Orphan. I am convinced that with an experienced partner like Eagle, we will succeed in making Landiolol available to patients in the U.S. as well," stated Georg Fischer, Chief Executive Officer of AOP Orphan.

The management of rapid heart rate (tachycardia) in critically ill patients can be quite complicated regardless of the underlying cause, which may include shock, arrhythmias, heart failure, and the postoperative setting. Beta blockers, also known as beta-adrenergic blocking agents, are a class of drugs that works by blocking the neurotransmitters norepinephrine and epinephrine from binding to receptors. These neurotransmitters contribute to the development of tachycardia. The β-1 receptor beta blockers are used frequently in critical care settings to manage tachycardia; however, the available β-1 beta blockers in the U.S. also can have the unwanted effects of decreasing the contractility, or muscle strength, of the heart, and of lowering blood pressure.

Landiolol has the potential to become a cornerstone therapy in the management of these patients. It is ultra short acting, with a rapid on and off effect that allows clinicians to balance heart rate control and blood pressure more precisely. In addition, it predominantly affects heart rate without much effect on cardiac contractility and blood pressure. The Company believes that clinicians will welcome Landiolol as a key therapeutic tool for the more precise management of tachycardia in the critical care setting.

There are additional clinical settings for which Landiolol has the potential to improve patient management. Enrollment is under way in Europe for a trial of Landiolol in patients with tachycardia and septic shock, and importantly, the product is also being studied in a pediatric population, for whom no beta-blocker drug products are approved in the U.S. for ventricular rate control. The U.S. FDA has tentatively agreed that this study could form the basis for initial pediatric study plan ("iPSP") for a future submission to FDA.

"We believe that we can expedite and prepare a compelling submission for approval of this important cardioprotective therapeutic," stated Judith Ng-Cashin, MD, Chief Medical Officer of Eagle Pharmaceuticals

Terms of the Agreement

The agreement is subject to regulatory clearance. Under the terms of the agreement, Eagle will make an upfront payment of $5 million, followed by additional payments upon regulatory approval(s) and based upon commercial sales. The agreement is subject to regulatory clearance.