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Aeglea BioTherapeutics Announces Pricing of $120 Million Public Offering

On April 28, 2020 Aeglea BioTherapeutics, Inc. (Nasdaq:AGLE), a clinical-stage biotechnology company developing next-generation human enzyme therapeutics as disruptive solutions for rare and other high-burden diseases, reported the pricing of an underwritten public offering of 11,652,830 shares of its common stock at a price to the public of $4.75 per share (Press release, Aeglea BioTherapeutics, APR 28, 2020, View Source [SID1234556679]). In addition, and in lieu of common stock, Aeglea is offering to certain investors pre-funded warrants to purchase up to an aggregate of 13,610,328 shares of common stock at a purchase price of $4.7499 per pre-funded warrant, which represents the per share public offering price for the common stock less the $0.0001 per share exercise price for each such pre-funded warrant. The aggregate gross proceeds from this offering are expected to be approximately $120 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by Aeglea. The offering is expected to close on or about April 30, 2020, subject to customary closing conditions. Aeglea has also granted the underwriters a 30-day option to purchase up to an additional 3,789,473 shares of common stock in connection with the public offering. All of the securities are being offered by Aeglea.

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J.P. Morgan Securities LLC, Evercore Group L.L.C. and Piper Sandler & Co. are acting as joint book-running managers in the offering. JonesTrading Institutional Services LLC and Needham & Company, LLC are acting as co-managers.

Aeglea intends to use the net proceeds from the offering, together with its existing cash resources, to advance the clinical development of pegzilarginase through its Phase 3 PEACE trial and BLA submission, advance its Phase 1/2 clinical trial and prepare for a potential Phase 3 trial of ACN00177 for Homocystinuria, and the remainder to fund continued research and development, manufacturing, commercialization infrastructure and for working capital and general corporate purposes.

The securities described above are being offered by Aeglea pursuant to a registration statement on Form S-3 previously filed and declared effective by the Securities and Exchange Commission (SEC). The offering is being made only by means of the written prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement and accompanying base prospectus relating to and describing the terms of the offering have been filed with the SEC and are available on the SEC’s website at www.sec.gov. Copies of the preliminary prospectus supplement and accompanying base prospectus, and when available, the final prospectus supplement and accompanying base prospectus may also be obtained from J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204 or by email at [email protected]; Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 36th Floor, New York, NY 10055, by telephone at (888) 474-0200, or by e-mail at: [email protected]; or Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, Attention: Prospectus Department, by telephone at (800) 747-3924 or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any securities of Aeglea, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

Constellation Pharmaceuticals to Host Conference Call to Discuss First Quarter 2020 Results

On April 28, 2020 Constellation Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported that it will host a conference call at 8:00 AM EDT on May 6, 2020, to discuss its first quarter 2020 results and progress in its clinical programs (Press release, Constellation Pharmaceuticals, APR 28, 2020, http://ir.constellationpharma.com/news-releases/news-release-details/constellation-pharmaceuticals-host-conference-call-discuss-first [SID1234556678]). The event will be webcast live and can be accessed on the Investor Relations section of Constellation’s website at View Source To participate in the live question-and-answer session, please dial (877) 473-2077 (domestic) or (661) 378-9662 (international) and refer to conference ID 4372778.

Moleculin Approved to Accelerate European Clinical Trial

On April 28, 2020 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates, reported that it is now authorized by the Polish Department of Registration of Medicinal Products known as URPL to accelerate the Phase 1 dose escalation portion of its clinical trial of Annamycin for the treatment of acute myeloid leukemia (AML) (Press release, Moleculin, APR 28, 2020, View Source [SID1234556677]).

Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)

The URPL has allowed an amendment to the Annamycin clinical trial protocol, which among other things, includes an increase in the dose escalation increment between cohorts from 30 mg/m2 to 60 mg/m2. The clinical trial is currently recruiting for the 240 mg/m2 cohort, so this amendment allows the next cohort to increase to 300 mg/m2, assuming all requirements for safety are met with the 240 mg/m2 cohort.

"Now that we have begun to demonstrate the absence of any cardiotoxicity associated with Annamycin," commented Walter Klemp, Chairman and CEO of Moleculin. "We believe we can and should move more aggressively to establish the maximum tolerated dose, or MTD, for Annamycin. This authorization now sets the stage to accelerate the dose escalation process. Thus far, even though we’ve seen promising activity from Annamycin, our dosing levels are still sub-therapeutic. Based on prior clinical experience with Annamycin, the 300 mg/m2 dosing level will be our first opportunity to test Annamycin at what we expect will be therapeutic levels."

Blueprint Medicines Announces Top-line Results from Phase 3 VOYAGER Trial of
Avapritinib versus Regorafenib in Patients with Advanced Gastrointestinal Stromal Tumor

On April 28, 2020 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported top-line results from the Phase 3 VOYAGER clinical trial of avapritinib versus regorafenib in patients with locally advanced unresectable or metastatic gastrointestinal stromal tumor (GIST) (Press release, Blueprint Medicines, APR 28, 2020, View Source [SID1234556676]). The VOYAGER trial did not meet the primary endpoint of an improvement in progression-free survival (PFS) for avapritinib versus regorafenib. Top-line safety data for avapritinib were consistent with those previously reported.

Blueprint Medicines plans to continue to commercialize AYVAKIT (avapritinib) in the United States for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and seek marketing approval for avapritinib for the treatment of this patient population in additional geographies. Blueprint Medicines continues to anticipate a decision from the European Commission on its marketing authorization application for the treatment of adults with PDGFRA D842V mutant GIST in the third quarter of 2020. Based on the top-line VOYAGER data, the company plans to discontinue further development of avapritinib in GIST beyond PDGFRA exon 18 mutant GIST.

"While we are disappointed by the outcome of the VOYAGER trial, we are deeply grateful to the patients, investigators and clinical site staff who contributed to the completion of this global study. We hope these data will reveal important insights to improve the scientific understanding of the disease and inform future innovations in GIST, and we are committed to sharing the results at a future medical meeting," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "At Blueprint Medicines, we strive to advance science, building on successes and learning from setbacks, to create new medicines for patients with difficult-to-treat cancers and rare diseases. With a deep portfolio of precision therapies and a strong financial position, we will continue to advance our pipeline with clear near-term priorities in systemic mastocytosis and RET-altered cancers."

Top-line Data from Phase 3 VOYAGER Trial

The VOYAGER trial evaluated the efficacy and safety of avapritinib (N=240) versus regorafenib (N=236) in patients with third- or fourth-line GIST.

Avapritinib showed a median PFS of 4.2 months compared to 5.6 months for regorafenib. The difference in median PFS between the avapritinib and regorafenib groups was not statistically significant. The overall response rate was 17 percent for the avapritinib group and 7 percent for the regorafenib group.

Avapritinib was generally well-tolerated with most adverse events reported as Grade 1 or 2. Top-line safety results were consistent with previously reported data, and no new safety signals were observed.

Additional analyses of the VOYAGER trial results are ongoing, and Blueprint Medicines plans to present the data at a future medical meeting.

Financial Guidance

Based on its current operating plans, Blueprint Medicines continues to expect that its existing cash, cash equivalents and investments together with anticipated product revenues but excluding any additional potential option fees, milestone payments or other payments under its collaboration or license agreements, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the second half of 2022. Anticipated savings from the discontinuation of further development of avapritinib in non-PDGFRA exon 18 mutant GIST indications is expected to offset any previously forecast revenues from those indications through 2022.

As of December 31, 2019, Blueprint Medicines had cash, cash equivalents and investments of $548.0 million. In addition, Blueprint Medicines received $308.4 million in estimated net proceeds from its January 2020 follow-on public offering.

Conference Call Information

Blueprint Medicines will host a live webcast today beginning at 8:00 a.m. ET to discuss the top-line data from the VOYAGER trial. To access the live call, please dial (855) 728-4793 (domestic) or (503) 343-6666 (international), and refer to conference ID 2207088. A webcast of the conference call will be available in the Investors & Media section of Blueprint Medicines’ website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

About the VOYAGER Trial

VOYAGER was a global, open-label, randomized, Phase 3 trial designed to evaluate the efficacy and safety of avapritinib versus regorafenib in patients with third- or fourth-line GIST. Patients were randomized 1:1 to receive either avapritinib (300 mg once daily dosing) or regorafenib (160 mg once daily dosing for three out of every four weeks) at multiple sites in the United States, Canada, European Union, Australia and Asia. The primary efficacy endpoint was PFS by blinded, independent central radiology review, based on modified Response Evaluation Criteria in Solid Tumors version 1.1 (mRECIST 1.1 criteria) for GIST. For more information about the VOYAGER trial, please visit www.clinicaltrials.gov (ClinicalTrials.gov Identifier: NCT03465722).

About AYVAKIT (avapritinib)

AYVAKIT (avapritinib) is a kinase inhibitor approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. AYVAKIT is the first precision therapy approved to treat a genomically defined population of patients with GIST and the only highly active treatment for PDGFRA exon 18 mutant GIST. The FDA granted Breakthrough Therapy Designation to avapritinib for the treatment of unresectable or metastatic GIST harboring the PDGFRA D842V mutation. For more information, visit AYVAKIT.com.

Avapritinib is not approved for the treatment of any other indication in the U.S. or any other jurisdiction by the FDA or any other health authority.

Blueprint Medicines is developing avapritinib globally for the treatment of advanced, smoldering and indolent systemic mastocytosis (SM). The FDA granted Breakthrough Therapy Designation to avapritinib for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.

Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of avapritinib and certain other drug candidates in Mainland China, HongKong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for avapritinib in the rest of the world.

Affimed Reports 2019 Financial Results and Recent Operational Progress

On April 28, 2019 Affimed N.V. (Nasdaq: AFMD), a clinical stage biopharmaceutical company committed to giving patients back their innate ability to fight cancer, reported financial results for the year ended December 31, 2019 and provided an update on clinical and corporate progress (Press release, Affimed, APR 28, 2020, View Source [SID1234556675]).

"Our clinical trials currently remain active, and we are working closely with our participating physicians and clinical sites to minimize the impact on further progress as they focus on addressing the evolving COVID-19 global health crisis," said Dr. Adi Hoess, Affimed’s CEO. "During these unprecedented and challenging times, our top priority continues to be supporting our employees, patients and our healthcare partners, while ensuring the continuity of our operations."

Development Program Updates

Affimed remains committed to continuing its development programs but acknowledges the impact from the evolving COVID-19 pandemic on clinical studies, including potential delays in patient enrollment. The company plans to update expected timing of milestones for its clinical studies after there is more clarity on the duration and magnitude of the impact from the COVID-19 pandemic.

AFM13 (CD30/CD16A)

Patient enrollment is ongoing in the Phase 2 registration-directed study of AFM13 as a monotherapy in relapsed or refractory patients with CD30-positive peripheral T cell lymphoma (pTCL). Affimed has successfully activated 42 clinical study sites across nine countries. To ensure patient safety and to avoid imposing additional burdens on healthcare systems already strained by the COVID-19 pandemic, the company has taken the decision to temporarily pause enrollment in the exploratory cohort of patients with CD30-positive relapsed or refractory transformed mycosis fungoides.

In March 2020, enrollment of all 15 patients was completed in an investigator-sponsored Phase 1b/2a study of AFM13 in patients with relapsed or refractory CD30-positive lymphoma with cutaneous manifestation led by Columbia University. Previously reported data from the study confirmed single-agent activity of AFM13, with an objective response rate (ORR) of 50% (5 of 10 patients).

Affimed is in close collaboration with the University of Texas MD Anderson Cancer Center (MDACC) to initiate an investigator-sponsored Phase 1 study to investigate the combination of AFM13 with allogeneic NK cells. MDACC intends to administer a stable complex of AFM13 pre-mixed with cord blood-derived allogeneic NK cells in different doses (numbers of pre-loaded NK cells) to patients with relapsed/refractory CD30-positive lymphoid malignancies.

AFM24 (EGFR/CD16A)

The first patient was successfully dosed in a first-in-human Phase 1/2a clinical trial of AFM24. The study is an open-label, non-randomized, multi-center, multiple ascending dose escalation/expansion study to evaluate AFM24 as monotherapy in patients with advanced solid malignancies known to be EGFR-positive and whose disease has progressed after treatment with previous anticancer therapies.

Preclinical Pipeline Update

Affimed continues to progress AFM28 and AFM32 as preclinical research currently remains unimpacted by the COVID-19 pandemic.

Genentech Collaboration Update

In November 2019, Genentech exercised its final option for an exclusive target under the ongoing, multi-program strategic oncology collaboration agreement to develop and commercialize novel NK cell engager-based immunotherapeutics generated from Affimed’s ROCK platform to treat multiple cancers. Affimed received a payment from Genentech in an undisclosed amount based on this achievement. Affimed and Genentech

continue to progress multiple programs under the ongoing strategic, multi-target collaboration.

Management Appointments

Dr. Andreas Harstrick joined Affimed as Chief Medical Officer (CMO) in March 2020 and Dr. Arndt Schottelius joined the company as Chief Scientific Officer (CSO) in April 2020. Dr. Harstrick brings a track record of running successful clinical trials that have led to regulatory approvals, deep oncology expertise, and a proven ability to lead large clinical organizations. Dr. Schottelius brings extensive innate immunity expertise, R&D leadership and a successful record of advancing and translating discovery research into preclinical and clinical development.

COVID-19 Impact Mitigation

COVID-19 has significantly impacted the global healthcare system, including the conduct of clinical trials as medical institutions prioritize the treatment of those afflicted with COVID-19. Affimed has taken several important actions to balance the commitment to treat cancer patients and to mitigate potential health and safety risks posed by the COVID-19 pandemic, while maintaining continuity of its operations and preserving financial flexibility for the future.

Full Year 2019 Financial Highlights

Cash, cash equivalents and current financial assets totaled €104.1 million as of December 31, 2019 compared to €108.8 million as of December 31, 2018. During the fourth quarter of 2019, the company raised €29.5 million in net proceeds, after deducting underwriting discounts and commissions and estimated offering expenses, from a completed public offering. Based on its current operating plan and assumptions, Affimed anticipates that its cash, cash equivalents and current financial assets as of December 31, 2019 will support operations at least into the first half of 2022.

Net cash used in operating activities was €29.1 million for the twelve months ended December 31, 2019, compared to net cash from operating activities of €49.4 million for the twelve months ended December 31, 2018. The net cash from operating activities in 2018 includes an initial upfront payment and committed funding of €83.2 million ($96.0 million) from the strategic collaboration Affimed entered into with Genentech Inc. in August 2018.

Total revenue was €21.4 million for the year ended December 31, 2019 compared to €23.7 million for the year ended December 31, 2018. Revenue in both years is attributable primarily to the recognition of revenue from the Genentech collaboration in the respective years.

Research and development (R&D) expenses were €43.8 million for the year ended December 31, 2019, compared to €35.1 million for the year ended December 31, 2018. The increase was primarily related to higher expenses for startup activities for the AFM13 registration-directed study in pTCL, manufacturing activities for AFM13 clinical study material, and early stage development and discovery activities.

General and administrative (G&A) expenses were €10.3 million for the year ended December 31, 2019, compared to €9.6 million for the year ended December 31, 2018. In 2019, G&A expenses were primarily related to personnel expenses and legal, consulting and audit costs.

Net loss was €32.4 million, or €0.50 per common share, for the year ended December 31, 2019, compared to a net loss of €19.5 million, or €0.32 per common share, for the year ended December 31, 2018.

Additional information regarding these results is included in the notes to the consolidated financial statements as of December 31, 2019 and "Item 5. Operating and Financial Review and Prospects," which will be included in Affimed’s Annual Report on Form 20-F as filed with the U.S. Securities and Exchange Commission (SEC).

Note on International Financial Reporting Standards (IFRS)

Affimed prepares and reports the consolidated financial statements and financial information in accordance with IFRS as issued by the International Accounting Standards Board. None of the financial statements were prepared in accordance with Generally Accepted Accounting Principles in the United States. Affimed maintains its books and records in Euro.

Conference Call and Webcast Information

Affimed will host a conference call and webcast today, Tuesday, April 28, 2020 at 8:30 a.m. Eastern time to discuss the company’s 2019 financial results and recent corporate developments. The conference call will be available via phone and webcast. To access the call, please dial +1 (877) 870-9135 for U.S. callers, or +44 (0) 2071 928338 for international callers, and reference passcode 7978047 approximately 15 minutes prior to the call.

A live audio webcast of the conference call will be available in the "Webcasts" section on the "Investors" page of the Affimed website at View Source, and a replay of the webcast will be accessible at the same link for 30 days following the call.