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 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with cancer, reported an Issue Notification for patent US 11,098,005 titled "METHODS FOR PRODUCING (6S,15S)-3,8,13,18- TETRAAZAICOSANE-6,15-DIOL" (Press release, Panbela Therapeutics, AUG 9, 2021, View Source [SID1234586104]). This patent, developed in collaboration with Syngene International Ltd., an integrated research, development, and manufacturing services company, claims a novel process for the production of our lead investigational product SBP-101, reduces the number of synthetic steps for its production from seventeen to six, and provides patent coverage to 2039.

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Jennifer K. Simpson, PhD, MSN, CRNP President & Chief Executive Officer of Panbela Therapeutics, commented, "We’re excited to receive this U.S. patent issuance, a landmark event for the company. This patent, covering a shorter synthesis of SBP-101, provides many benefits including: 1) the ability to manufacture product with a reduced lead time 2) quicker access to drug supply facilitating expansion into additional indications and 3) enables a scalable, efficient and cost-effective manufacturing process for future commercialization. We applaud the dedicated efforts of our valued long-term partner Syngene International Ltd. in helping us achieve this important goal."

Jonathan Hunt, Managing Director and Chief Executive Officer, Syngene International said, "We congratulate Panbela for this milestone achievement. Our partnership with them dates back to 2013 and since then we have collaborated on multiple projects. In this case, streamlining the production process of an investigational product is a core expertise in our Development Services division. If the drug is approved, a simpler production process means that commercialization will be easier and the drug will reach patients more quickly."

Dr. Simpson added, "This process developed utilizes a pharmaceutical starting material that is widely available, assuring the company of drug supply moving forward. The Company expects to continue innovation and patent portfolio building as it develops its clinical programs".

About SBP-101
SBP-101 is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. The molecule has shown signals of tumor growth inhibition in clinical studies of US and Australian metastatic pancreatic cancer patients, suggesting potential complementary activity with an existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, SBP-101 has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Recently observed serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the current Panbela sponsored clinical trial provides support for continued evaluation of SBP-101 in a randomized clinical trial. For more information, please visit View Source .

On August 9, 2021 Biofrontera AG (NASDAQ: BFRA; Frankfurt Stock Exchange: B8F) (the "Company"), an international biopharmaceutical company, reported preliminary, unaudited revenue for the month of July 2021 (Press release, Biofrontera, AUG 9, 2021, View Source [SID1234586103]).

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The Company’s preliminary, unaudited revenue from product sales in July 2021 amounted to approximately EUR 1,570 thousand, compared to EUR 1,523 thousand in July 2020, an increase of 3%.

Preliminary revenues from product sales in the US were around EUR 1,026 thousand compared to EUR 795 thousand in July 2020, an increase of 29%. In Germany, revenues from product sales amounted to approximately EUR 357 thousand, compared to EUR 572 thousand in July 2020, a decrease of 38%. In this context, the relatively high comparative sales in July 2020 in Germany resulted from catch-up effects due to the weak previous months caused by the pandemic. In the rest of Europe, the Company generated product sales of around EUR 187 thousand, compared to EUR 156 thousand in July 2020, a plus of 20%.

Due to the pandemic, the monthly and year-to-date sales development is also compared with sales in 2019 for increased transparency. As such, an increase of 9% in July 2021 for total product sales was achieved in all markets compared to July 2019. In the USA, product sales increased by about 15% compared to July 2019. In July 2021, sales were up by around 6% in Germany while sales revenue in the remaining European markets decreased by approximately 11% compared to July 2019.

Compared to 2019, product sales in all markets were down by around 3% compared to sales in the period January to July 2021. Year-to-date sales in 2021 increased by around 23% in Germany and by 22% in the rest of the European market compared to the same period in 2019. Revenue in the US market was down 13%, mainly due to weak sales in January, February, and the first half of March this year due to the pandemic, as well as the lack of or lower sales of Aktipak and Xepi compared to the period January to July 2019.

On August 9, 2021 Navrogen, Inc., a privately held preclinical biotechnology company, reported that it has raised $3.0M from an equity financing to support its Humoral Immuno-Oncology (HIO) discovery platform and preclinical therapeutic pipeline activities (Press release, Navrogen, AUG 9, 2021, View Source [SID1234586102]). HIO is a process by which tumors produce factors that suppress a patient’s humoral immune response against malignant tissues. The major use of proceeds will focus on advancing the company’s lead HIO-refractory, anti-mesothelin NAV-001 antibody drug conjugate program and its NK cell activator platforms that specifically address HIO suppressed cancers and immune-mediated diseases. The financing was led by Tellus BioVentures along with existing investors. As part of the financing, Lonnie Moulder, Managing Member of Tellus, and Allan Bedwick, will join the Navrogen Board of Directors.

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"We are pleased to have Tellus BioVentures lead this round of funding as we take steps to further validate and advance our pipeline agents towards clinical development," stated Nicholas Nicolaides, Ph.D., Chief Executive Officer at Navrogen. "With Tellus, we gain additional access to professionals with deep biopharmaceutical operational and drug development experience that coupled with our extensive internal expertise will strengthen the pursuit of our value-creating objectives to deliver innovative medicines to patients with HIO-positive cancers and other immune-related disorders."

Lonnie Moulder, Managing Member of Tellus BioVentures, added, "We are thrilled to support Navrogen and its accomplished founding team in their efforts to achieve their platform and pipeline goals. Navrogen has discovered a unique translational approach for developing a new class of compounds to address immune-mediated diseases, including various cancer types".

On August 9, 2021 Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) ("Eagle" or the "Company") reported financial results for the three and six months ended June 30, 2021 (Press release, Eagle Pharmaceuticals, AUG 9, 2021, View Source [SID1234586080]).

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Business and Recent Highlights:

Entered into a licensing agreement for the U.S. commercial rights to Landiolol, a leading hospital emergency use product in Europe and Japan. Landiolol is currently approved in Europe for the treatment of non-compensatory sinus tachycardia and tachycardic supraventricular arrhythmias. Eagle 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.
Advanced vasopressin program and continue to expect a commercial launch prior to year-end:
Responded to the Complete Response Letter ("CRL") for its Abbreviated New Drug Application ("ANDA") for vasopressin received from FDA in February 2021;
FDA maintained Priority Review for the Company’s ANDA for vasopressin and assigned a GDUFA date of December 15, 2021;
Patent trial against Endo Par Innovation Company, LLC took place on July 7, 2021; Court ruling expected around mid-September.
Approval of TREAKISYM (bendamustine) ready-to-dilute ("RTD") formulation, in combination with rituximab for treatment of relapsed or refractory diffuse large B-cell lymphoma ("r/r DLBCL") received from the Pharmaceuticals and Medical Devices Agency ("PMDA") in Japan. This represents a meaningful extension of Eagle’s bendamustine franchise and is expected to significantly increase the market opportunity for bendamustine;
Filing of TREAKISYM rapid infusion ("RI") (50ml) liquid formulation with the PMDA in Japan. Eagle expects RTD, and RI, if approved, formulations to generate approximately $20-$25 million of combined royalty and milestone revenue;
Appointed former FDA Official and Public Health Expert Dr. Luciana Borio to its Board of Directors; and
Continue to pursue additional in-licensing and acquisition opportunities to broaden Eagle’s pipeline and revenue streams.
Financial Highlights

Second Quarter 2021

Total revenue for Q2 2021 was $48.1 million, compared to $41.9 million in Q2 2020, primarily reflecting higher product sales of BELRAPZO and RYANODEX, partially offset by lower product sales of BENDEKA.

Q2 2021 net income was $3.6 million, or $0.28 per basic and $0.27 per diluted share, compared to net loss of $0.3 million, or ($0.02) per basic and diluted share in Q2 2020.
Q2 2021 adjusted non-GAAP net income was $12.4 million, or $0.95 per basic and $0.93 per diluted share, compared to adjusted non-GAAP net income of $8.0 million, or $0.59 per basic and $0.57 per diluted share, in Q2 2020.
Cash and cash equivalents were $108.7 million, net accounts receivable was $52.7 million, and debt was $30 million as of June 30, 2021.
"We had a strong and productive quarter on multiple fronts and have laid the foundation for important growth drivers going forward. We made good progress with vasopressin during the quarter and continue to believe that we will be able to launch that important product before year-end. We are just months away from our February 2022 PEMFEXY launch, which allows us an initial period of exclusivity in a billion-dollar market. With the potential for an additional royalty and milestone revenue stream of $20-$25 million from the expanding TREAKISYM franchise in Japan, next year could be a record earnings year for Eagle," stated Scott Tarriff, Chief Executive Officer of Eagle Pharmaceuticals.

"The licensing agreement for Landiolol represents Eagle’s first new chemical entity and is a true catalyst in reshaping our company as we evolve from a specialty pharmaceutical company into a mainstream pharmaceutical company, with a vibrant pipeline of products. Landiolol solidifies our hospital and critical care product portfolio, and we plan to leverage our current sales force with little additional infrastructure costs to promote the product. We are also pursuing other such opportunities to build value for the company and look forward to providing updates," concluded Tarriff.

Second Quarter 2021 Financial Results

Total revenue for the three months ended June 30, 2021 was $48.1 million, compared to $41.9 million for the three months ended June 30, 2020.

Q2 2021 BELRAPZO product sales were $7.6 million, compared to $4.1 million in Q2 2020.

Q2 2021 RYANODEX product sales were $7.9 million, compared to $4.7 million in Q2 2020.

Royalty revenue was $28.5 million in the second quarter of 2021, compared to $27.6 million in the second quarter of 2020. BENDEKA royalties were $27.8 million in the second quarter of 2021, compared to $27.5 million in the second quarter of 2020. A summary of total revenue is outlined below:

Gross Margin was 78% during the second quarter of 2021, as compared to 69% in the second quarter of 2020. The increase in gross margin for the second quarter of 2021 was driven by revenue mix.

R&D expense was $9.9 million for the second quarter of 2021, compared to $7.1 million in the second quarter of 2020. The increase is largely attributable to development cost for vasopressin of $1.5 million, RYANODEX related projects of $0.8 million and PEMFEXY of $0.6 million, partially offset by $0.5 million decrease in development activity related to fulvestrant. Excluding stock-based compensation and other non-cash and non-recurring items, R&D expense during the second quarter of 2021 was $9.2 million.

SG&A expenses in the second quarter of 2021 totaled $16.6 million compared to $18.0 million in the second quarter of 2020. The decrease is primarily related to lower stock compensation expense and marketing spend. Excluding stock-based compensation and other non-cash and non-recurring items, second quarter 2021 SG&A expense was $12.4 million.

Net income for the second quarter of 2021 was $3.6 million, or $0.28 per basic and $0.27 per diluted share, compared to net loss of $0.3 million, or ($0.02) per basic and diluted share, in the second quarter of 2020, due to the factors discussed above.

Adjusted non-GAAP net income for the second quarter of 2021 was $12.4 million, or $0.95 per basic and $0.93 per diluted share, compared to adjusted non-GAAP net income of $8.0 million or $0.59 per basic and $0.57 per diluted share in the second quarter of 2020. For a full reconciliation of adjusted non-GAAP net income to the most comparable GAAP financial measures, please see the tables at the end of this press release.

2021 Expense Guidance

R&D spend in 2021, on a non-GAAP basis, is expected to be $34-$38 million, as compared to $27.8 million in 2020.
SG&A spend in 2021, on a non-GAAP basis, is expected to be $52-$56 million, as compared to $50.9 million in 2020.
The guidance provided in this section represents forward-looking information, and actual results may vary. Please see the risks and assumptions referred to in the Forward-Looking Statements section of this press release.

Liquidity

As of June 30, 2021, the Company had $108.7 million in cash and cash equivalents plus $52.7 million in net accounts receivable. The Company had $30 million in outstanding debt. Therefore, as of June 30, 2021, the Company had net cash plus receivables of $131.4 million.

In the second quarter of 2021, the Company purchased $2.9 million of its common stock as part of its $160.0 million Share Repurchase Program. From August 2016 through June 30, 2021, the Company has repurchased $211.2 million of its common stock.

Conference Call

As previously announced, Eagle management will host its second quarter 2021 conference call as follows:

www.eagleus.com, under the "Investor + News" section

A replay of the conference call will be available for one week after the call’s completion by dialing 888-562-2815 (US) or 402-220-7352 (International) and entering conference call ID EGRXQ221. The webcast will be archived for 30 days at the aforementioned URL.