On October 28, 2021 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and financial results for the third quarter ending September 30, 2021 (Press release, PTC Therapeutics, OCT 28, 2021, View Source [SID1234592117]).

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"The stellar growth of our DMD franchise is remarkable." said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "I continue to be impressed by the team’s performance. The commercial performance, along with the milestones achieved in Brazil, and the continued advancements in our pipeline, with initiating a fifth registration-directed trial, allows us continued value creation for all of our stakeholders."

Key Third Quarter and Other Corporate Updates:

The Duchenne muscular dystrophy (DMD) franchise grew 39% over the third quarter of 2020, demonstrating PTC’s continued strong commercial performance. Quarterly net product revenue for the franchise was $114 million in the third quarter of 2021.
Translarna (ataluren) revenue growth was driven by geographic expansion and new patient identification.
Emflaza (deflazacort) revenue growth was driven primarily by new patient starts and maintained high compliance.
Evrysdi(risdiplam) continues to show strong global uptake. The first commercial sale of Evrysdi in Japan occurred in August 2021, resulting in a $10 million milestone payment from Roche to PTC with $325 million in sales-based milestones remaining. Evrysdi is a product of a collaboration between PTC, Roche and the SMA Foundation.
Waylivra (volanesorsen) was approved by the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), as the first treatment for familial chylomicronemia syndrome (FCS) in Brazil.
Tegsedi (inotersen) has successfully received Category 1 classification from CMED (Drug Market Regulation Chamber) in Brazil. Category 1 classification is given to innovative treatments that provide greater efficacy than current standards of care and allows for pricing in line with international markets.
Third Quarter Clinical Updates:

The APHENITY registration-directed Phase 3 trial for PTC923 for phenylketonuria (PKU) has been initiated with results expected by the end of 2022.
PTC continues to make progress in three additional ongoing registration-directed clinical studies:
The MIT-E Phase 2/3 vatiquinone trial for mitochondrial disease associated seizures with results anticipated in the third quarter of 2022.
The MOVE-FA Phase 3 vatiquinone trial for Friedreich ataxia with results anticipated in 2023.
Enrollment in the FITE19 Phase 2/3 emvododstat trial in patients with COVID-19 is expected to be completed by year end 2021.
The Phase 1 healthy volunteer study of PTC518 for Huntington’s disease met the objectives in the third quarter of this year. The Phase 2 study in patients with Huntington’s disease is expected to be initiated by the end of 2021.
The Committee for Medicinal Products for Human Use (CHMP) opinion on the PTC-AADC gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency is expected in the fourth quarter of 2021.
For the Biologics License Application (BLA) for AADC deficiency, PTC expects to submit the BLA in the first quarter of 2022.
Third Quarter 2021 Financial Highlights:

Total revenues were $138.7 million for the third quarter of 2021, compared to total revenues of $118.4 million for the third quarter of 2020.
Total revenue includes net product revenue across the commercial portfolio of $115.6 million and royalty and collaboration revenue of $23.1 million for the third quarter of 2021, compared to net product revenues of $82.7 million and royalty and collaboration revenues of $35.7 million for the third quarter of 2020.
Translarna net product revenues were $67.2 million for the third quarter of 2021, compared to $43.4 million for the third quarter of 2020. These results reflect an increase in net product sales in existing markets as well as continued geographic expansion.
Emflaza net product revenues were $47.1 million for the third quarter of 2021, compared to $38.5 million for the third quarter of 2020. These results reflect new patient prescriptions, high compliance, and fewer discontinuations.
Roche reported Evrysdi 2021 year to date sales of approximately CHF 396 million, resulting in year-to-date royalty revenue of $33.3 million to PTC. During the third quarter of 2021, the first commercial sale of Evrysdi in Japan triggered a $10 million milestone payment from Roche to PTC, which was reported as collaboration revenue.
U.S. GAAP (generally accepted accounting principles) research and development (R&D) expenses were $130.8 million for the third quarter of 2021, compared to $93.0 million for the third quarter of 2020. The increase reflects additional investment in research programs and advancement of the clinical pipeline.
Non-GAAP R&D expenses were $117.8 million for the third quarter of 2021, excluding $13.0 million in non-cash stock-based compensation expense, compared to $83.8 million for the third quarter of 2020, excluding $9.2 million in non-cash stock-based compensation expense.
GAAP selling, general and administrative (SG&A) expenses were $69.3 million for the third quarter of 2021, compared to $57.8 million for the third quarter of 2020. The increase reflects our continued investment to support commercial activities including expanding our commercial portfolio.
Non-GAAP SG&A expenses were $56.4 million for the third quarter of 2021, excluding $12.8 million in non-cash stock-based compensation expense, compared to $50.3 million for the third quarter of 2020, excluding $7.6 million in non-cash stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was $10.8 million for the third quarter of 2021, compared to $8.4 million for the third quarter of 2020. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.
Net loss was $133.6 million for the third quarter of 2021, compared to net loss of $69.7 million for the third quarter of 2020.
Cash, cash equivalents and marketable securities was $867.9 million at September 30, 2021, compared to $1.1 billion at December 31, 2020.
Shares issued and outstanding as of September 30, 2021 were 70,665,010.
PTC Updates Full Year 2021 Guidance as Follows:

PTC now anticipates net product revenues for the DMD franchise for the full year 2021 to be between $400 and $420 million from previous guidance of $370 and $390 million.
PTC now anticipates GAAP R&D and SG&A expense for the full year 2021 to be between $815 and $835 million from previous guidance of $825 and $855 million.
PTC now anticipates Non-GAAP R&D and SG&A expense for the full year 2021 to be between $715 and $735 million, excluding estimated non-cash, stock-based compensation expense of $100 million, from previous guidance of $725 and $755 million.
Non-GAAP Financial Measures:

In this press release, the financial results and financial guidance of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the Company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the tables below.

Today’s Conference Call and Webcast Reminder:

PTC will host a conference call to discuss the third quarter of 2021 corporate updates and financial results today at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 7064479. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.

On October 28, 2021 Prescient Therapeutics (PTX) reported it is poised to reach value-adding milestones across its anti-cancer programs after wrapping up the September quarter with $14.8 million in cash (Press release, Prescient Therapeutics, OCT 28, 2021, View Source;utm_medium=rss&utm_campaign=prescient-therapeutics-asxptx-logs-timely-progress-across-anti-cancer-programs-in-q3 [SID1234592116]).

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Based in Melbourne, Prescient is a clinical-stage oncology company developing personalised medical approaches to cancer, including targeted and cellular therapies.

Finances
The health stock reported quarterly costs, including investments in clinical studies of its PTX-100 and PTX-200 targeted therapies; pre-clinical development of its OmniCAR platform; and the Cell Therapy Enhancement Program.

Net cash outflows for the quarter clocked in at $1.47 million, with around $782,000 invested in research and development across Australia and the United States.

Notably, PTX said it had retained a strong cash position and was headed towards value-creating milestones on the back of timely progress made across its anti-cancer programs.

OmniCAR update
Prescient continued to work on its OmniCAR technology during the September period.

OmniCAR is a universal immune receptor platform that enables controllable T-cell activity and multi-antigen targeting with a single cell product.

PTX said it had received positive in-silico results from immunogenicity testing of the platform’s key binding components, SpyTag and SpyCatcher, which was designed to test the immune response against the therapy and its overall safety profile.

The results reportedly showed low immunogenicity — equal to circulating human antibodies.

The company said the results de-risked the platform ahead of future in-house and external collaborations.

Cell Therapy Enhancement program
Meanwhile, Prescient’s Cell Therapy Enhancement program progressed towards a number of important pre-clinical milestones under the guidance of the research team at Melbourne’s Peter MacCallum Cancer Centre.

While the particulars of this work remain undisclosed for competitive reasons, PTX said the nature and outcomes of the research held significant possibilities for cancer treatments.

Further updates
Notably, Prescient also reported "successful" results from Phase 1b of its PTX-100 basket trial, in which it said the therapy showed an excellent safety profile.

PTX-100 will now progress to an expansion cohort study focused on T cell lymphomas, with potential for a subsequent registration study.

Lastly, the company welcomed brain cancer expert Professor Donald M. O’Rourke to its Scientific Advisory Board.

Despite the update, shares in Prescient Therapeutics were down 1.85 per cent to 26.5 cents each at 2:30 pm AEDT.

On October 28, 2021 Pascal Biosciences Inc. ("Pascal" or the "Company") (TSXV: PAS) (OTC: PSCBF) (FSE: 6PB-FF) reported the following actions to continue the development of the Company’s PAS-403 and PAS-393 patented molecules for development of cancer therapeutics prior to commencing clinical trials and for general working capital to support this direction (Press release, Pascal Biosciences, OCT 28, 2021, View Source [SID1234592115]).

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The Company announces a non-brokered private placement financing of up to 9,000,000 units (each a "Unit") of securities at a price of $0.10 per Unit for aggregate gross proceeds of up to $900,000.00 (the "Offering"). Each Unit will be comprised of one (1) common share and one (1) non-transferable common share purchase warrant, with each whole warrant entitling the holder to purchase one additional common share at a price of $0.15 for two years from the closing date of the private placement. The exercise term of the warrant will be accelerated in the event the closing price of the Shares is above $0.20 for ten consecutive days and will be exercisable for a period of 15 days from the date of receipt by investors of a Notice of Acceleration. Finder’s fees of up to 10% may be paid.

All securities issued will be subject to a four month holding period which will expire on the date that is four months and one day from the date of issue.

On October 28, 2021 Pan Cancer T B.V., a biotech spin-off from the Erasmus MC dedicated to the discovery and development of novel TCR-T therapies against solid tumors, reported the closing of an extended seed investment round (Press release, Pan Cancer T, OCT 28, 2021, https://pancancer-t.com/2021/10/28/pan-cancer-t-extends-seed-financing-round-with-additional-funding-from-existing-shareholders-and-new-investor-thuja-capital/ [SID1234592114]). All current shareholders participated in the round, with Thuja Capital joining as a new investor. The undisclosed proceeds add to the initial seed capital financing announced earlier this year. The Company’s syndicate of experienced life science venture capital investors now includes Van Herk Ventures, Swanbridge Capital, and Thuja Capital as well as founding shareholder Erasmus MC.

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Katrien Reynders-Frederix, CEO of Pan Cancer T, said: "Despite certain successes of adoptive cell therapies like the Chimeric Antigen Receptor (CAR) T cell therapy, the vast majority of solid cancers remains refractory to such treatments. TCR-T cell therapy is a novel, promising option that has demonstrated significant clinical benefits in patients with various solid tumors. Thanks to the extended seed round and the continuous support of our investors we will be able to further advance our lead program and strengthen our pre-clinical assets. Next, we will be preparing to raise a Series A financing round to advance our lead program into the clinic."

Michel Briejer, Investment Manager at Thuja Capital added: "Since we identified Pan Cancer T as an investment opportunity, we have been deeply impressed by the quality of the science, the team and its advisers. We are thrilled to join the shareholder base and take a seat on the Supervisory Board to help and support Pan Cancer T towards the success it is bound for."

Dharminder Chahal, Investment Manager at Van Herk and Swanbridge Capital, said: "I am pleased to see that Pan Cancer T made such a progress within only one year after its foundation. I am convinced that the company will continue this high pace of development through this additional investment."

Prof. Chris Bangma, Professor of the Department of Urology at Erasmus University Medical Centre in Rotterdam, stated: "Erasmus MC valorizes the development of novel therapeutic treatment options for its patients. The results of immune therapy in various tumors are beyond expectations. Many patients may benefit from the carefully designed new immunologic approaches and evaluation in the clinic. Hence, Erasmus MC is pleased to support Pan Cancer T on this journey."

On October 28, 2021 OPKO Health, Inc. (NASDAQ: OPK) reported that business highlights and financial results for the three months ended September 30, 2021 (Press release, Opko Health, OCT 28, 2021, View Source [SID1234592113]).

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Business Highlights

Health Canada approved NGENLA (somatrogon) injection for pediatric growth hormone deficiency. NGENLA is a once-weekly, long-acting recombinant human growth hormone, for the long-term treatment of pediatric patients who have growth failure due to an inadequate secretion of endogenous growth hormone (growth hormone deficiency, or GHD). Please click here to view Pfizer Canada’s news release issued earlier today. Global regulatory applications for somatrogon continue to advance with action dates in the U.S., Europe and Japan expected during the first half of 2022.

BioReference Laboratories (BRL) processed approximately 2.2 million COVID-19 PCR tests in the third quarter of 2021. In addition, during the quarter BRL performed approximately 158,100 COVID-19 serology tests to measure SARS-CoV-2 antibody levels and currently has significant additional capacity. In August 2021 Rite Aid, the U.S. Department of Health and Human Services, and BRL announced a "Back to School" COVID-19 testing program offering students at New York State public schools the opportunity for free COVID-19 testing prior to or at the start of the 2021-2022 school year. Students will be tested using highly accurate RT-PCR laboratory-based COVID-19 tests.

BRL acquired the U.S. Ariosa centralized laboratory prenatal testing business from Roche. Ariosa’s noninvasive prenatal screening (NIPS) test, the Harmony Prenatal Test, is one of the most widely studied tests utilized in prenatal screening. This test has been performed in more than 1.5 million patients. The acquisition of Ariosa will complement the current NIPS offering at GenPath, BRL’s specialty health division.

Completed enrollment in Phase 2 trial evaluating RAYALDEE as a treatment for symptomatic COVID-19 outpatients. The U.S. trial, "A Randomized, Double-Blind Placebo-Controlled Study to Evaluate the Safety and Efficacy of RAYALDEE (calcifediol) Extended-release Capsules to Treat Symptomatic Patients Infected with SARS-CoV-2," completed final enrollment with 171 subjects, including some with stage 3 or 4 chronic kidney disease who are at higher risk for developing more severe illness. Topline data are expected later this year.

Formed a joint venture with LeaderMed Group to develop, manufacture and commercialize oxyntomodulin and Factor VIIa-CTP in China and other Asian territories. Under the terms of the agreements, OPKO granted the joint venture exclusive rights to develop, manufacture and commercialize OPK88003, an oxyntomodulin analog being developed for the treatment of obesity and diabetes, and Factor VIIa-CTP, a novel long-acting coagulation factor being developed to treat hemophilia, in exchange for a 47% ownership interest in the joint venture. LeaderMed will be responsible for funding the joint venture’s operations, development and commercialization efforts. OPKO retains full rights to oxyntomodulin and Factor VIIa-CTP in all other geographies.

Executed exclusive worldwide agreement with CAMP4 Therapeutics Corporation (CAMP4) for the development, manufacture and commercialization of therapeutics utilizing the AntagoNAT technology. This technology is an oligonucleotide platform developed under OPKO CURNA. CAMP4 has prioritized OPKO’s lead AntagoNAT compound to progress into clinical trials for the treatment of Dravet syndrome. Under the terms of the agreement, OPKO received an upfront payment and shares of privately held CAMP4. In addition, OPKO will be eligible to receive up to $93.5 million and additional shares upon the achievement of certain development and sales milestones for products developed from this technology and associated intellectual property. CAMP4 will also pay OPKO double-digit royalties on product sales.
Third Quarter Financial Results

Consolidated: Consolidated total revenues for the third quarter of 2021 were $385.8 million compared with $428.1 million for the comparable period of 2020. Operating income for the third quarter of 2021 increased 72% to $37.8 million compared with $21.9 million, for the comparable period of 2020. Net income for the third quarter of 2021 was $28.7 million, or $0.04 per diluted share, compared with $23.7 million, or $0.04 per diluted share, for the comparable period of 2020.

Diagnostics: Revenue from services in the third quarter of 2021 decreased to $340.1 million from $382.5 million in the prior-year period, primarily due to a decrease in COVID-19 testing volume, which was partially offset by an improvement in COVID-19 test reimbursement and an increase in clinical and genomic test revenue. Total costs and expenses were $320.5 million in the third quarter of 2021 compared with $346.4 million in the third quarter of 2020, resulting in operating income of $19.7 million compared with $46.2 million in the 2020 period. The decrease in operating income is primarily due to a decline in COVID-19 test volume and an increased level of investment in our commercial organization for our base business and digital health activities resulting in increased professional fees and personnel expenses. During the third quarter of 2020, BioReference received a $10.0 million non-recurring grant under the CARES Act.

Pharmaceuticals: Revenue from products in the third quarter of 2021 increased almost 29% to $36.9 million compared with $28.7 million in the third quarter of 2020, with the increase primarily attributable to accelerating growth within OPKO’s international pharmaceutical businesses. Total prescriptions for RAYALDEE in the third quarter of 2021 decreased to approximately 11,500 from approximately 16,700 in the third quarter of 2020. Revenue from sales of RAYALDEE in the third quarter of 2021 was $8.5 million compared with $8.1 million in the prior-year period, with the increase reflecting a higher net realized price as a result of lower Medicare Part D rebates. Revenue from the transfer of intellectual property was $8.8 million in the third quarter of 2021 compared with $6.8 million for the 2020 period. The increase reflects the CAMP4 and LeaderMed agreements partially offset by a decrease in revenue related to the Pfizer transaction. Total costs and expenses were $17.0 million in the third quarter of 2021 compared with $49.9 million in the prior-year period, primarily due to a $31.5 million gain on the sale of assets to Horizon Therapeutics plc for OPKO’s sterile-fill-finish manufacturing facility in Ireland. Operating income was $28.6 million in the third quarter of 2021 compared with an operating loss of $14.4 million in the third quarter of 2020.

Cash and equivalents: Cash, cash equivalents and marketable securities were $148.6 million as of September 30, 2021. In addition, the Company has $64.3 million availability under its line of credit with JP Morgan.
CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update, discuss third quarter financial results and answer questions during a conference call and live audio webcast today beginning at 4:30 p.m. Eastern time. Participants are requested to pre-register for the conference call using the link here. Upon pre-registering, participants will receive dial-in numbers, an event passcode and a unique registrant ID to gain immediate access to the call and bypass the live operator. Participants may pre-register at any time, including up to and after the start of the call. Alternatively, please dial (888) 869-1189 or (706) 643-5902 and use conference ID 6958207

To access the live call via webcast, please click on the link OPKO 3Q21 Results Conference Call. Individual investors and investment community professionals who do not plan to ask a question during the call’s Q&A session are encouraged to listen to the call via the webcast.

For those unable to listen to the live conference call, a replay can be accessed for a period of time on OPKO’s website at OPKO 3Q21 Results Conference Call. A telephone replay will be available beginning approximately two hours after the completion of the conference call. To access the replay, please dial (855) 859-2056 or (404) 537-3406, and use conference ID 6958207.