On November 3, 2021 Protagonist Therapeutics, Inc. (Nasdaq: PTGX) ("Protagonist" or "the Company") reported its financial results for the third quarter ended September 30, 2021 and provided a corporate update (Press release, Protagonist, NOV 3, 2021, View Source [SID1234594227]).

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"We are excited to share the substantial progress made thus far in all of our clinical programs, highlighted by the presentation of proof-of-concept data on rusfertide in hereditary hemochromatosis at the upcoming AASLD meeting, and the new rusfertide data in polycythemia vera, which will be presented by the year’s end," said Dinesh V. Patel, Ph.D., President and Chief Executive Officer. "We were very pleased that the FDA reached a swift resolution regarding the clinical hold placed on rusfertide, and we are in the process of resuming all rusfertide clinical studies. Additionally, we are highly encouraged with the rate of enrollment in the Phase 2 study of our oral alpha-4-beta-7 integrin antagonist PN-943, for ulcerative colitis, an indication with a large patient population and unmet treatment need. We maintain our guidance of a data readout in Q2 2022, and in anticipation of this, we are excited that Dr. Scott Plevy has joined our team as Executive Vice President and Therapeutic Head, Gastroenterology. As a renowned gastroenterologist, Dr. Plevy will oversee the clinical development of PN-943 and other future programs focused on gastrointestinal diseases. Finally, our oral IL-23 receptor antagonist program, in partnership with Janssen, continues to make demonstrated progress, with PN-235 now advancing into a Phase 2 study in psoriasis in early 2022."

Third Quarter 2021 Recent Developments and Upcoming Milestones

Rusfertide: Subcutaneous Injectable Hepcidin Mimetic for Polycythemia Vera (PV) and Other Blood Disorders

On October 11, 2021, Protagonist announced that the U.S. Food and Drug Administration (FDA) removed the full clinical hold on rusfertide’s clinical studies after the Company provided the FDA with all requested information, including the individual patient clinical safety reports, updated the investigator brochure and patient informed consent forms, conducted a comprehensive review of the most recent safety database, and added new safety and stopping rules in the study protocols. This was in response to the Company receiving a communication from the FDA that Protagonist’s clinical studies for rusfertide had been placed on clinical hold on September 17, 2021. Protagonist has been working closely with study investigators and clinical trial sites to resume enrollment and dosing of patients in ongoing clinical trials with rusfertide.
An abstract highlighting new, preliminary data from a Phase 2 proof-of-concept study in hereditary hemochromatosis (HH) has been selected for oral presentation by Dr. Kris Kowdley, from the Liver Institute Northwest in Seattle. These data will be presented on November 13, 2021 at The Liver Meeting 2021, hosted by the American Association for the Study of Liver Diseases (AASLD).
The Company intends to report updated data from the Phase 2 study of rusfertide in polycythemia vera (PV) by the end of 2021.
The Company is on track to initiate the Phase 3 study of rusfertide in PV in Q1 2022.
The Company resolved its collaboration agreement dispute with Zealand Pharma, reducing future development and sales milestone payments and royalties owed to Zealand for rusfertide under the companies’ 2012 collaboration agreement. Under the new terms of the agreement, future development and sales milestone payments (other than $2.5 million in near-term milestones) and royalties for rusfertide have been reduced by 50 percent. Milestones and royalty payments will be due for sales and milestones achieved by either Protagonist or any future rusfertide licensee or partner. Protagonist will also make a $1.5 million payment to Zealand in August 2022.
PN-943: Oral, gut-restricted, alpha-4-Beta-7 Integrin Antagonist for Ulcerative Colitis

We are continuing to see steady progress in the enrollment and execution of the Phase 2 IDEAL study of PN-943 in ulcerative colitis. This study includes a 12-week induction period and a 40-week open label extension. Topline data from the 12-week induction period is expected in the second quarter of 2022.
Scott Plevy, M.D., renowned expert in translational and clinical research in gastroenterology and immunology, was appointed Executive Vice President and Therapeutic Head, Gastroenterology, and he will oversee clinical operations and development of Protagonist’s ongoing and future programs in gastrointestinal diseases, including PN-943.
Oral IL-23 Receptor Antagonists (collaboration between Janssen Biotech, Inc. and Protagonist)

Following a pre-specified interim analysis criteria, a portfolio decision was made to stop further development of the first-generation IL-23 receptor antagonist (IL-23R) candidate PTG-200 (JNJ-67864238), in favor of continued development of the two second generation candidates PN-235 (JNJ-77242113) and PN-232 (JNJ-75105186) with superior product profiles. In particular:
The Phase 1 study of PN-235 is completed, and a Phase 2 study in psoriasis is anticipated to initiate in early 2022.
The Phase 1 study with PN-232 is under progress with study completion expected by mid-2022.
Additional development in IBD is expected to initiate in 2022.
Protagonist will earn a $25 million milestone in connection with the initiation of the first Phase 2 study of a second-generation candidate, and a $10 million milestone in connection with the initiation of the second Phase 2 study of a second-generation candidate. Protagonist remains eligible for up to approximately $900 million in development-related milestone payments, in addition to the $87.5M in milestones already earned.
Protagonist received a $7.5 million milestone payment from Janssen, triggered by the completion of the clinical data collection Phase 1 activities for PN-235 (JNJ-2113).
Third Quarter 2021 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of September 30, 2021, were $352.5 million. The Company expects current cash, cash equivalents and marketable securities to be sufficient to fund its planned operating and capital expenditures through 2024.
License and Collaboration Revenue: License and collaboration revenues were $10.3 million and $18.7 million for the three and nine months ended September 30, 2021, respectively, as compared to $13.1 million and $23.0 million reported for the same periods of 2020. The Company recognized $8.0 million as a cumulative catch-up amount during the three months ended September 30, 2021, following the amendment of its collaboration agreement for the development of IL-23 receptor antagonist assets with Janssen Biotech. This cumulative catch-up was primarily the result of an acceleration of our cumulative performance completed under our obligation, following the amendment to the collaboration which reduced the remaining services the Company is responsible to conduct. We are nearing completion of our remaining services to be provided to Janssen under the collaboration, in particular, both the ongoing Phase 1 trials in PN-235 and PN-232, are expected to be complete in the fourth quarter of 2021 and second quarter 2022, respectively. Revenue for the prior year’s third quarter of 2020 also included an estimate update for services completed versus remaining services to be performed under the Janssen collaboration agreement which accelerated revenue recognition.
Research and Development ("R&D") Expenses: R&D expenses for the three and nine months ended September 30, 2021, were $37.0 million and $87.6 million, respectively, as compared to $16.0 million and $55.0 million, respectively, for the same periods of 2020. The increases were primarily due to additional costs associated to advancing our clinical trials with our pipeline assets rusfertide and PN-943, as well as our second-generation IL-23 receptor antagonist assets under the Janssen collaboration (PN-235 and PN-232). The increases also relate to higher research spending and employee related costs, including stock-based compensation expenses following recent hiring in support of our advancing research and development programs. Following the amendment to the Zealand collaboration agreement we also recorded $4.0 million in the quarter ended September 30, 2021 in R&D expense related to these collaboration payments.
General and Administrative ("G&A") Expenses: G&A expenses for the three and nine months ended September 30, 2021, were $7.3 million and $19.9 million, respectively, as compared to $4.9 million and $13.6 million for the same periods of 2020. The increases were primarily related to professional fees, insurance costs and employee compensation related expenses, including stock-based compensation expenses, supporting the growth in our operations.
Stock Based Compensation ("SBC") Expenses: SBC expenses for the three and nine months ended September 30, 2021, were $4.8 million and $11.4 million, respectively, as compared to $1.8 million and $5.9 million, respectively, for the same periods of 2020. SBC increases included in R&D and G&A expenses are primarily attributable to the addition of new employees and related stock grants in support of the Company’s continued growth coupled with the increase in the Company’s stock price at grant date which is used in the SBC expense calculation.
Net Loss: The third quarter 2021 net loss was $33.8 million, or a net loss of $0.70 per share, and the nine months ended September 30, 2021, net loss was $88.6 million, or a net loss of $1.94 per share, compared to the third quarter of 2020 net loss of $7.8 million, or a net loss of $0.21 per share, and the nine months ended September 30, 2020, net loss of $47.3 million, or a net loss of $1.45 per share.

On November 3, 2021 QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported that results for the third quarter and first nine months of 2021 (Press release, Qiagen, NOV 3, 2021, View Source [SID1234594226]).

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In Q3 2021, net sales rose 11% (+10% at constant exchange rates, CER) to $535 million, which was well above the outlook for CER sales to be at the same level as in the year-ago period. Key drivers were a 48% CER gain in sales of the QuantiFERON-TB test to $79 million over Q3 2020 and solid growth in DNA sample technologies products. Also supporting the performance was a stronger-than-expected demand for COVID-19 testing solutions. Diluted earnings per share (EPS) were $0.57 in Q3 2021, up from $0.07 per share in the same period of 2020. Adjusted diluted EPS was $0.58 ($0.58 CER), which was above the outlook for $0.52-0.53 CER and unchanged from Q3 2020.

"QIAGEN’s results for the third quarter of 2021 confirm that our business continues to build strong momentum, especially our non-COVID product groups that delivered 17% CER growth," said Thierry Bernard, Chief Executive Officer of QIAGEN N.V. "We are on track to reach our 2021 sales targets for our five pillars of growth while crossing a significant milestone of more than $2 billion in net sales. Our performance during 2021 has underscored the relevancy of QIAGEN in the molecular research and testing market, while we stand ready to address the volatile trends in COVID-19 testing."

Roland Sackers, Chief Financial Officer of QIAGEN N.V., said: "We have upgraded our full-year outlook for 2021 to reflect the stronger-than-expected results in the third quarter of 2021. Using the boost from the COVID-19 pandemic demand, as well as the momentum in our non-COVID product groups, we are making significant progress on initiatives to support the business expansion in the coming years thanks to our record operating cash flow combined with a commitment to disciplined capital allocation. This has given us the flexibility to make investments to further accelerate our five pillars of growth while increasing returns to shareholders, such as through the recently completed $100 million share repurchase program."

Please find the full press release incl. tables here

Investor presentation and conference call

A conference call is planned for Thursday, November 4, 2021, at 14:30 Central European Time (CET) / 9:30 Eastern Daylight Time (EDT). A live audio webcast will be made available in the investor relations section of the QIAGEN website, and a replay will also be made available after the event. A presentation is available at View Source as of Thursday, November 4, 2021, at 12:30 Central European Time (CET) / 7:30 Eastern Daylight Time (EDT).

On November 3, 2021 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel small molecules to treat and improve the lives of patients with genetically defined cancers, reported financial results for the third quarter of 2021 (Press release, Biomea Fusion, NOV 3, 2021, View Source [SID1234594225]).

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"The Third Quarter was a historic one for the company as it advanced its first investigational drug into a clinical stage program. BMF-219, our internally discovered and developed small molecule, has shown broad anti-cancer activity pre-clinically and we now have the opportunity to explore this potential in patients in need." said Thomas Butler, Biomea’s CEO and Chairman of the Board. "In September, we received FDA clearance of our IND for BMF-219, and we are now working diligently to initiate a Phase I study for the treatment of relapsed or refractory acute leukemia. We look forward to providing clinical development updates as we deepen our understanding of menin’s role in various cancers and the potential impact of irreversible inhibition for these patient populations. Our vision for this company has always been to develop a suite of novel covalent inhibitors that can act as single agent therapies or in combination, together, to build best-in-class medicine for patients. We have executed on this promise by hiring world-class drug hunters, and developers, and by custom designing and opening a state-of-the-art Research Center. We now have our first molecule ready for the clinic, and a number of exciting programs advancing through pre-clinical development."

Mr. Butler continued, "As we move BMF-219 into the clinic, we intend to also focus on leveraging our novel chemistry across multiple indications. To that end, we anticipate announcing our next pipeline candidate in the first half of 2022."

Business Highlights

FDA clearance of IND for First-in-Human Phase I Clinical Trial of BMF-219. In September 2021, Biomea received FDA clearance of its IND for BMF-219, an irreversible menin inhibitor for the treatment of relapsed or refractory acute leukemias including those with an MLL/KMT2A gene rearrangement or NPM1 mutation.
Preclinical studies underway for DLBCL and multiple myeloma (MM) as potential indications for BMF-219. Biomea is conducting preclinical studies to demonstrate the potential for BMF-219 in genetically defined patient subsets of DLBCL and MM.
Pathway validation studies of irreversible menin inhibition in diabetes. Biomea is continuing preclinical studies to explore the potential of its irreversible menin inhibitor approach for the treatment of type 2 diabetes. The company plans to report findings from these studies in the fourth quarter of 2021.
Launch of Biomea Innovation Center; scale-up of R&D organization. Biomea recently opened the Biomea Innovation Center with laboratory space and a dedicated R&D team focused on leveraging the company’s FUSION platform to generate a broad portfolio of next generation covalent inhibitors.
"Our CEO’s vision for the company is to build a fully sustainable, world-class R&D organization, which mines the deep potential of our FUSION platform and irreversible binding small molecule approach. With our recently opened Biomea Innovation Research Center and our continued scale-up of an extremely talented, forward-and creative-thinking scientific team, we will aggressively interrogate our irreversible binding approach against known and validated biological targets with the goal of rapidly advancing a broad portfolio of therapies designed to deliver enhanced efficacy and safety for patients. Thanks to our successful IPO and considering our current programs’ quarterly expenses, we expect our cash balance to sustain our operations well into 2024," concluded Ramses Erdtmann, Biomea’s COO and President.

Financial Highlights

Third Quarter 2021 Year to Date Financial Results

Biomea reported a net loss attributable to common stockholders of $26.9 million for the first nine months of 2021, compared to a net loss of $1.8 million for the same period in 2020.
Research and development expenses were $16.9 million for the first nine months of 2021, compared to $1.3 million for the same period in 2020. The increase of $15.6 million was primarily due to an increase in personnel-related expenses, as well as an increase in pre-clinical development costs, including manufacturing and external consulting, related to the IND-enabling studies for BMF-219.
General and administrative expenses were $10.0 million for the first nine months of 2021, compared to $0.5 million for the same period in 2020. The increase of $9.5 million was primarily due to higher personnel-related expenses and other corporate costs to support the Company’s public company status.
As of September 30, 2021, the Company had cash, cash equivalents, restricted cash, and investments of $191.9 million.
About Acute Myeloid Leukemia (AML)

AML is the most common form of acute leukemia in adults and represents the largest number of annual leukemia deaths in the U.S. and Europe. AML originates within the white blood cells in the bone marrow and can rapidly move to the blood and other parts of the body, including the lymph nodes, spleen, and central nervous system. Approximately 30,000 people in the U.S. and Europe are diagnosed with AML each year, and the five-year overall survival rate in adults roughly 29%. Among patients with relapsed/refractory disease, the need is greatest, as the overall survival is approximately 3 to 9 months. It is estimated that upwards of 45% of AML patients have menin dependent genetic drivers (MML-r or NPM1).

About BMF-219

BMF-219 is an irreversibly binding inhibitor of menin, a protein that is known to play an essential role in oncogenic signaling in genetically defined leukemias. Preclinically, BMF-219 has demonstrated robust downregulation of key leukemogenic genes in addition to menin itself (via MEN1) in well-established MLLr AML cell lines. Additionally, BMF-219 has shown efficacy in multiple in vivo and in vitro models of acute leukemias. BMF-219 will be evaluated in a first-in-human trial in patients with relapsed or refractory acute leukemia with MLL/KMT2A gene rearrangement or NPM1 mutation.

On November 3, 2021 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage precision oncology company focused on developing targeted drugs to inhibit frontier targets that drive and sustain RAS-addicted cancers, reported that it will report financial results for the third quarter 2021 on November 10, 2021 after market close (Press release, Revolution Medicines, NOV 3, 2021, View Source [SID1234594224]). The company will host a conference call and webcast at 4:30 p.m. Eastern Time during which members of Revolution Medicines’ senior management team will discuss financial results for the quarter and review recent corporate developments.

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To listen to the live webcast, or access the archived webcast, please visit the "Events & Presentations" page of Revolution Medicines’ website at: View Source Following the live webcast, a replay will be available on the Company’s website for at least 14 days.

To listen to the live conference call, please dial (833) 423-0425 or (918) 922-3069 and request the Revolution Medicines call (conference ID: 9757524).

On November 3, 2021 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), reported financial results for the three months ended September 30, 2021 and provided an update on key milestones (Press release, Lexicon Pharmaceuticals, NOV 3, 2021, View Source [SID1234594223]).

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"We are on track for submission around year-end of our new drug application for sotagliflozin for the treatment of heart failure in patients with type 2 diabetes," said Lonnel Coats, Lexicon’s chief executive officer. "Momentum continues to build for the adoption of SGLT inhibitors into the first-line heart failure treatment regimen, as most recently evidenced by the new clinical practice guidelines issued in late August by the European Society of Cardiology (ESC). We believe that sotagliflozin’s unique mechanism of action as a dual SGLT1 and SGLT2 inhibitor offers differentiating advantages in this emerging market, with a particular opportunity in heart failure with preserved ejection fraction (HFpEF), and that a primary point of engagement will be with specialists treating patients who have been recently hospitalized for worsening heart failure, which was the cornerstone of our SOLOIST study. We are building our launch readiness team in preparation for potentially bringing sotagliflozin to patients suffering from heart failure and living with type 2 diabetes next year."

Third Quarter Highlights

Sotagliflozin

ESC issued new treatment guidelines adding SGLT inhibitors to the standard of care for the prevention and treatment of heart failure. Sotagliflozin was specifically recommended for the primary prevention of heart failure in type 2 diabetes patients with risk factors for its development and the treatment of heart failure in patients with type 2 diabetes, and was also noted in the guidelines in the context of worsening heart failure.
Additional analyses of sotagliflozin data from the SOLOIST and SCORED Phase 3 clinical studies were presented at the ESC Congress 2021 highlighting the benefits seen in reduced heart failure hospitalizations, myocardial infarction and stroke.
LX9211

Patient enrollment continued in two ongoing Phase 2 clinical studies of LX9211 for the treatment of diabetic peripheral neuropathic pain and the treatment of post-herpetic neuralgia. Results from these clinical studies are currently expected in the first half of 2022.
Corporate

Craig B. Granowitz, M.D., Ph.D. joined Lexicon as senior vice president and chief medical officer, with a deep and successful history in cardiovascular medicine. Dr. Granowitz will lead the build-out of Lexicon’s medical affairs organization as the company prepares for the potential launch of sotagliflozin for the treatment of heart failure in patients with type 2 diabetes.
Third Quarter 2021 Financial Highlights

Revenues: Revenues for the three months ended September 30, 2021 were negligible as compared to $6.6 million for the corresponding period in 2020, primarily due to the absence of product revenues as a result of Lexicon’s sale of its XERMELO product and related assets to TerSera Therapeutics LLC during the third quarter of 2020.

Research and Development (R&D) Expenses: Research and development expenses for the three months ended September 30, 2021 decreased to $15.7 million from $40.1 million for the corresponding period in 2020, primarily due to decreases in external clinical development costs related to sotagliflozin resulting from the completion of clinical studies.

Selling, General and Administrative (SG&A) Expenses: Selling, general and administrative expenses for the three months ended September 30, 2021 decreased to $7.3 million from $12.0 million for the corresponding period in 2020, primarily due to lower salaries and benefits costs as a result of reductions in personnel in September 2020 and lower marketing expenses, all primarily associated with the sale of XERMELO and related assets during the third quarter of 2020.

Net Loss: Net loss for the three months ended September 30, 2021 was $23.1 million, or $0.16 per share, as compared to net income of $82.6 million, or $0.71 per diluted share, in the corresponding period in 2020. The three months ended September 30, 2020 included a gain of $132.8 million from the sale of XERMELO and related assets to TerSera. For the three months ended September 30, 2021 and 2020, net income included non-cash, stock-based compensation expense of $2.7 million and $1.9 million, respectively.

Cash and Investments: As of September 30, 2021, Lexicon had $120.9 million in cash and investments, as compared to $152.3 million as of December 31, 2020.

Conference Call and Webcast Information

Lexicon management will hold a live conference call and webcast today at 5:00 pm ET / 4:00 pm CT to review its financial and operating results and to provide a general business update. The dial-in number for the conference call is 888-645-5785 (U.S./Canada) or 970-300-1531 (international). The conference ID for all callers is 8564549. The live webcast and replay may be accessed by visiting Lexicon’s website at www.lexpharma.com/events. An archived version of the webcast will be available on the website for 14 days.

About the SOLOIST and SCORED Studies

SOLOIST was a multi-center, randomized, double-blinded, placebo-controlled Phase 3 study evaluating the cardiovascular efficacy of sotagliflozin versus placebo when added to standard of care in 1,222 patients with type 2 diabetes who had recently been hospitalized for worsening heart failure. The primary endpoint was the total number of events comprised of deaths from cardiovascular causes, hospitalizations for heart failure, and urgent visits for heart failure in patients treated with sotagliflozin compared with placebo.

SCORED was a multi-center, randomized, double-blinded, placebo-controlled Phase 3 study evaluating the cardiovascular efficacy of sotagliflozin versus placebo when added to standard of care in 10,584 patients with type 2 diabetes, chronic kidney disease with eGFR of 25 to 60 ml per minute per 1.73 m2 of body-surface area, and risks for cardiovascular disease. The primary endpoint was the total number of events comprised of deaths from cardiovascular causes, hospitalizations for heart failure, and urgent visits for heart failure in patients treated with sotagliflozin compared with placebo.

Both SOLOIST and SCORED achieved their respective primary endpoints. Results from both studies were presented at the Late-Breaking Science Session of the American Heart Association (AHA) Scientific Sessions 2020 and simultaneously published in The New England Journal of Medicine (NEJM) in two separate articles titled: "Sotagliflozin in Patients with Diabetes and Recent Worsening Heart Failure" and "Sotagliflozin in Patients with Diabetes and Chronic Kidney Disease."