On May 4, 2021 Innate Pharma SA (Euronext Paris: IPH – ISIN: FR0010331421; Nasdaq: IPHA) ("Innate" or the "Company"), reported that the Company will hold a conference call on Thursday, May 11, 2021 at 3 p.m. CEST / 9 a.m. ET, to give an update on business progress during the first quarter of 2021 (Press release, Innate Pharma, MAY 4, 2021, View Source [SID1234579040]).

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Participating in the call will be Innate Pharma’s Chief Executive Officer Mondher Mahjoubi, MD, Chief Medical Officer Joyson Karakunnel, MD, MSc, FACP, and Chief Financial Officer Frédéric Lombard, MBA.

Details for the Virtual Event

The live webcast will be available at the following link:

View Source

Participants may also join via telephone to ask questions by registering in advance of the event at View Source Upon registration, participants will be provided with dial-in numbers, a direct event passcode and a unique registrant ID that they may use 10 minutes prior to the event start to access the call. Call reminders will also be sent to registered participants via e-mail the day prior to the event.

This information can also be found on the Investors section of the Innate Pharma website, www.innate-pharma.com. A replay of the webcast will be available on the Company website for 90 days following the event.

On May 4, 2021 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, reported positive top-line results from the Company’s GENESIS Phase 3 trial evaluating its lead clinical candidate, Motixafortide, in combination with granulocyte colony stimulating factor (G-CSF, the standard of care in this indication), for hematopoietic stem-cell mobilization for autologous bone marrow transplantation in multiple myeloma patients (Press release, BioLineRx, MAY 4, 2021, View Source [SID1234579067]).

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An analysis of data on all 122 enrolled patients (the intent to treat, or ITT, population) found highly statistically significant evidence across all primary and secondary endpoints favoring Motixafortide in addition to G-CSF, as compared to placebo plus G-CSF. In addition, the combination was found to be safe and well tolerated.

The primary endpoint of the study demonstrated a 4.9-fold increase (70.0% vs 14.3%; difference 54.6%; 95% CI 39.7-69.5%; p<0.0001) in the proportion of patients in the treatment arm, as compared to the control arm mobilizing ≥ 6 million CD34+ cells/kg in up to two apheresis sessions, and after only one administration of Motixafortide. This translates to an odds-ratio of 12.9.

The study also achieved its main secondary endpoint, demonstrating a 14.1-fold increase (67.5% vs 4.8%; difference 61.7%; 95% CI 49.5-73.8%; p<0.0001) in the proportion of patients in the treatment arm, as compared to the control arm, who mobilized ≥ 6 million CD34+ cells/kg in just one apheresis session. This translates to an odds-ratio of 56.0.

Other important data from the study include median number of CD34+ cells collected on the first day of apheresis (8.5 million in the treatment arm vs 1.5 million in the control arm) – a 5.6-fold increase. The addition of Motixafortide to G-CSF also allowed 88.3% of patients to undergo transplantation after only one apheresis session, compared to 10.8% in the G-CSF arm – an 8.2-fold increase. Engraftment endpoints, including the number of days needed for engraftment, success of engraftment and the durability of engraftment 100 days post-transplant, further support the study’s success.

"The results of the GENESIS study are extremely impressive, and all the more so when considering that almost 90% of the patients in the treatment arm proceeded to transplantation after only one apheresis session," stated John DiPersio, MD, Washington University School of Medicine, and lead investigator of the study. "This is a great achievement in alleviating the burden for the patients and reducing hospital resources. I believe these results make the combination of Motixafortide and G-CSF a very attractive candidate for use in all patients with multiple myeloma undergoing autologous stem-cell transplantation."

"These strikingly positive data significantly exceeded our expectations, and are truly transformational for our company," stated Philip Serlin, Chief Executive Officer of BioLineRx. "The statistical significance across all primary and secondary endpoints was consistent across twelve different sensitivity analyses. These results support our goal of becoming the standard of care for autologous bone-marrow transplantation, providing a strong clinical and pharmaco-economic advantage for its use, on top of G-CSF, in all transplant procedures.

"We are working aggressively to gain regulatory approval for Motixafortide in this transplant setting for multiple myeloma patients – with plans to make an NDA submission in the first half of next year – and we are also pressing forward to unlock the full potential of this therapy in this and other stem-cell mobilization indications. I would like to express our sincere thanks to the patients and investigators who participated in the study and enabled its great success," Mr. Serlin concluded.

Conference Call and Webcast Information
BioLineRx will hold a conference call today, Tuesday, May 4, 2021 at 10:00 a.m. EDT. To access the conference call, please dial +1-866-860-9642 from the US or +972-3-918-0644 internationally. The call will also be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

A replay of the conference call will be available for a limited time approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until May 6, 2021; please dial +1-888-295-2634 from the US or +972-3-925-5904 internationally.

About the GENESIS Trial
The GENESIS trial (NCT03246529) was initiated in December 2017. GENESIS was a randomized, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of Motixafortide and G-CSF, compared to placebo and G-CSF, for the mobilization of hematopoietic stem-cells for autologous transplantation in multiple myeloma patients. The primary objective of the study was to demonstrate that only one dose of Motixafortide on top of G-CSF is superior to G-CSF alone in the ability to mobilize ≥ 6 million CD34+ cells in up to two apheresis sessions. Additional objectives included time to engraftment of neutrophils and platelets and durability of engraftment, as well as other efficacy and safety parameters. Local laboratories and a central laboratory were used to determine CD34+ cell yields. For regulatory purposes, efficacy endpoints were calculated using the percentage of CD34+ cells determined by the central laboratory. The local laboratory values were used for all clinical decisions, including the number of apheresis days and the decision to proceed to transplantation.

On May 4, 2021 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and financial results for the first quarter ending March 31, 2021 (Press release, PTC Therapeutics, MAY 4, 2021, View Source [SID1234579090]).

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"Overall PTC has had a strong performance this quarter through all aspects of the company from discovery to commercial revenue," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "I would like to highlight the continued strong growth of the DMD franchise which has had one of our largest quarterly revenues to date. The other key milestone was the positive preliminary results in our PTC518 Huntington’s disease program demonstrating dose dependent lowering of the HTT mRNA. Analogous to the SMA program we are now well positioned with a clear path for success."

Key First Quarter and Other Corporate Updates:

The Duchenne muscular dystrophy (DMD) franchise had a total net product revenue of $90 million for Translarna (ataluren) and Emflaza (deflazacort) in the first quarter of 2021. This represents 32% growth over the first quarter of 2020 and one of PTC’s strongest quarterly commercial revenues to date.
Broader uptake due to new patients in existing geographies and geographic expansion drove Translarna growth.
Emflaza revenue growth was primarily due to increased new prescriptions, high compliance, and fewer discontinuations.
In March 2021, the European Medicines Agency (EMA) approved Evrysdi (risdiplam) in the European Union (EU). The first sale of Evrysdi in this region was recorded the following day, triggering a $20 million milestone payment to PTC. Evrysdi is a product of a collaboration between PTC, Roche and the SMA Foundation.
Preliminary results from the PTC518 Phase 1 healthy volunteer trial demonstrated dose-dependent reduction of Huntington mRNA beyond the 30-50% target.
PTC received Gallup’s Don Clifton Strengths-Based Culture Award, which reflects the Company’s ongoing deep commitment to its employees.
First Quarter Clinical Updates:

PTC has multiple clinical trials ongoing, three of which are registration-directed clinical studies:
The MIT-E Phase 2/3 trial with vatiquinone for mitochondrial epilepsy with data anticipated in the third quarter of 2022.
The MOVE-FA Phase 3 trial with vatiquinone for Friedreich ataxia with data anticipated in 2023.
The FITE19 Phase 2/3 clinical trial for PTC299 in patients with COVID-19 with an expected data readout in the second half of 2021.
The second Bio-e compound, PTC857 healthy volunteer study was recently completed, and data will be communicated in the second quarter.
The registration-directed Phase 3 PTC923 phenylketonuria (PKU) trial, APHENITY, is expected to initiate in mid-2021.
The Committee for Medicinal Products for Human Use (CHMP) has requested a clock stop in the aromatic L-amino acid decarboxylase (AADC) deficiency review process to allow for completion of its pre-approval inspections, which were delayed due to COVID-19. The CHMP opinion is now anticipated in the third quarter of 2021.
Due to COVID-related surgical delays, the AADC-deficiency biologics license application submission to the U.S. Food and Drug Administration is anticipated to be delayed by at least one quarter.
First Quarter 2021 Financial Highlights:

Total revenues were $117.9 million for the first quarter of 2021, compared to total revenues of $68.3 million for the first quarter of 2020, a 32% increase. Total revenue includes net product revenue of $91.3 million and collaboration and royalty revenue of $26.7 million in the first quarter of 2021.
Translarna net product revenues were $46.5 million for the first quarter of 2021, compared to $40.5 million for the first quarter of 2020. These results reflect an increase in net product sales in existing markets as well as continued geographic expansion into new territories.
Emflaza net product revenues were $43.5 million for the first quarter of 2021, compared to $27.5 million for the first quarter of 2020. These results reflect new patient prescriptions, high compliance, and fewer discontinuations.
Roche reported Evrysdi first quarter 2021 sales of approximately CHF 80 million. During the first quarter of 2021, the first commercial sale of Evrysdi in the EU triggered a $20 million milestone payment to PTC, which was reported as collaboration revenue.
U.S. GAAP (generally accepted accounting principles) R&D expenses were $134.5 million for the first quarter of 2021, compared to $90.1 million for the first quarter of 2020. The increase in R&D expenses reflects costs associated with increased investment in research programs, and advancement of the clinical pipeline.
Non-GAAP R&D expenses were $120.8 million for the first quarter of 2021, excluding $13.7 million in non-cash, stock-based compensation expense, compared to $81.9 million for the first quarter of 2020, excluding $8.2 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $61.1 million for the first quarter of 2021, compared to $58.2 million for the first quarter of 2020. The increase in SG&A expenses was associated with entering into a long-term lease for the Hopewell facility that commenced on July 1, 2020.
Non-GAAP SG&A expenses were $49.1 million for the first quarter of 2021, excluding $12.0 million in non-cash, stock-based compensation expense, compared to $51.2 million for the first quarter of 2020, excluding $7.0 million in non-cash, stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was $0.1 million for the first quarter of 2021, compared to $0.9 million for the first 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 $128.6 million for the first quarter of 2021, compared to net loss of $112.7 million for the first quarter of 2020.
Cash, cash equivalents and marketable securities was $988.4 million at March 31, 2021, compared to $1.1 billion at December 31, 2020.
Shares issued and outstanding as of March 31, 2021 were 70,405,905.
PTC Reaffirms Full Year 2021 Guidance as Follows:

PTC anticipates net product revenues for the DMD franchise for the full year 2021 to be between $355 and $375 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2021 to be between $825 and $855 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2021 to be between $725 and $755 million, excluding estimated non-cash, stock-based compensation expense of $100 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 table below.

Today’s Conference Call and Webcast Reminder:

PTC will host a conference call to discuss the first 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 4292410. 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 May 4, 2021 Oncorus, Inc. (Nasdaq: ONCR), a viral immunotherapies company focused on driving innovation to transform outcomes for cancer patients, reported first quarter 2021 financial results and highlighted recent achievements and developments (Press release, Oncorus, MAY 4, 2021, View Source [SID1234579109]).

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"We began 2021 with strong momentum, announcing the buildout of our GMP manufacturing facility which is now well underway, and we continue to advance our ambitious goals on behalf of cancer patients," said Theodore (Ted) Ashburn, M.D., Ph.D., President and Chief Executive Officer of Oncorus.

Dr. Ashburn further commented, "We continue to enroll patients in a Phase 1 clinical trial of ONCR-177, our lead oncolytic Herpes Simplex Virus (oHSV) clinical candidate, and expect initial interim data later this year. We also anticipate nominating our first two synthetic viral RNA (vRNA) immunotherapy candidates in the first half of 2021. These candidates are comprised of vRNA coding for oncolytic viruses encapsulated within lipid nanoparticles, or LNPs – proprietary technologies developed by the Oncorus team. We have designed this novel approach to enable the systemic, repeat intravenous (IV) administration of viral immunotherapies, the so-called ‘holy grail’ of this modality, to date unattainable. We’re excited to introduce this breakthrough approach and discuss these candidates in more detail at an upcoming virtual investor event."

First Quarter 2021 and Recent Highlights

Enrolling Phase 1 clinical trial of ONCR-177. Oncorus is currently enrolling a Phase 1 clinical trial of its lead product candidate, ONCR-177, an intratumorally (iTu) administered oHSV viral immunotherapy being developed for multiple solid tumor indications. The Phase 1 open-label, multi-center, dose escalation and expansion clinical trial is designed to evaluate the safety and tolerability of ONCR-177. The trial will determine the recommended Phase 2 dose, as well as investigate ONCR-177’s preliminary anti-tumor activity, alone and in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), in patients with advanced and/or refractory cutaneous, subcutaneous or metastatic nodal solid tumors. Oncorus has an ongoing clinical trial collaboration with Merck involving KEYTRUDA and anticipates reporting interim data from the Phase 1 trial in the second half of 2021 through the second half of 2022.
Advancing lead Synthetic vRNA Immunotherapy Platform programs toward clinical candidate nomination. Oncorus continues to advance its lead synthetic, IV administered vRNA immunotherapy programs based on the Coxsackievirus A21 (CVA21) and the Seneca Valley Virus (SVV). The company expects to nominate clinical candidates for both programs in the first half of 2021. IV administration of viral immunotherapies is an attractive approach for improving the standard of care for many cancer patients because it allows for all tumors, including micro-metastases, to be directly treated. In addition, it allows for the potential treatment of certain tumors, such as those of the lung, that are less amenable to repeat iTu injection of anti-cancer therapies due to safety and feasibility considerations. Oncorus’ Synthetic vRNA Immunotherapy Platform includes a novel LNP delivery strategy designed to overcome the challenges caused by neutralizing antibodies, which have limited the efficacy of previous industry efforts to treat tumors utilizing IV administration of OVs.
Advancing second oHSV viral immunotherapy candidate, ONCR-GBM. Leveraging its oHSV Platform, Oncorus is pursuing ONCR-GBM to specifically target brain cancer, including glioblastoma multiforme (GBM). The company is utilizing its knowledge of microRNA expression to engineer a microRNA attenuation strategy to protect healthy brain tissue and select a combination of payloads intended to address the specific drivers of immune suppression in brain cancer. Oncorus plans to nominate its ONCR-GBM clinical candidate in the second half of 2021.
Announced buildout of Good Manufacturing Practice (GMP) viral immunotherapy clinical manufacturing facility. In January 2021, Oncorus announced the signing of a 15-year lease to build a state-of-the-art, 88,000 square foot GMP viral immunotherapy clinical manufacturing facility in Andover, Mass. The facility is intended to provide a comprehensive solution for Oncorus’ Chemistry, Manufacturing and Controls (CMC) development needs, enabling the manufacture, quality, control and supply of clinical-grade viral immunotherapies for investigational new drug (IND)-enabling and clinical studies. Oncorus anticipates the first phase of the facility’s buildout will be completed in late 2021, including process development and quality control, with GMP multi-product manufacturing capabilities and full operation commencing in early 2023.
Completed follow-on public offering. In February 2021, Oncorus completed an underwritten public offering of common stock, at a price of $19.00 per share, raising $57.0 million in aggregate gross proceeds.
First Quarter Financial Results

Cash and cash equivalents were $172.6 million as of March 31, 2021 compared to $130.3 million as of December 31, 2020.
Research and development expenses for the quarter ended March 31, 2021 were $8.4 million compared to $5.9 million for the corresponding quarter in 2020. The increase in research and development expenses was mainly attributable to increased rent expense related to the Company’s new manufacturing facility, increased personnel-related expenses, including stock-based compensation, driven by increased headcount and increased clinical trial costs for the Company’s ongoing Phase 1 clinical trial of ONCR-177.
General and administrative expenses for the quarter ended March 31, 2021 were $4.2 million compared to $2.1 million for the corresponding quarter in 2020. The increase in general and administrative expenses was primarily attributable to increases in personnel-related expenses, including stock-based compensation, driven by increased compensation and increased headcount and increased costs, such as insurance expense and professional and consultant expenses, related to operating as a public company.
Net loss attributable to common stockholders for the quarter ended March 31, 2021 was $12.7 million, or $0.53 per share, compared to a net loss attributable to common stockholders of $10.6 million, or $10.59 per share for the same quarter in 2020. The share and loss per share amounts in the first quarter of 2021 reflect the impact of the company’s IPO, which closed in October 2020, including the conversion of outstanding preferred stock into approximately 15.0 million shares of common stock.
Financial Guidance

Based upon its current operating plans and cash and cash equivalents, Oncorus expects to have sufficient capital to fund its operating expenses and capital expenditure requirements into late 2023.

On May 4, 2021 Mogrify Limited (Mogrify), a biopharmaceutical company aiming to transform the development of ex vivo cell therapies and pioneer a new class of in vivo reprogramming therapies, reported the second closing of its Series A financing of $17 million USD, bringing the total raised to $33 million USD in this round (Press release, Mogrify, MAY 4, 2021, View Source [SID1234579125]). The funding will support the advancement of Mogrify’s immuno-oncology and ophthalmology programs, as well as continued platform development and the exploration of cell reprogramming for novel therapeutic application.

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This second close of the Company’s Series A was led by Parkwalk Advisors and incorporates additional funding from strategic Corporate Pharma investor Astellas Venture Management as well as 24Haymarket, co-Founder of Abcam PLC, Dr Jonathan Milner, and Mogrify CEO, Dr Darrin M Disley OBE.

Proceeds from the financing will be used to advance Mogrify’s immuno-oncology and ophthalmology programs, inclusive of iPSC-derived allogeneic cell therapies targeting hematological and solid malignancies, as well as in vivo reprogramming therapies aiming to address retinal degeneration, a leading cause of blindness, through to IND-enabling studies.

Mogrify’s proprietary suite of platform technologies enable systematic identification of the key transcriptomic and epigenetic cell switches that drive the speed, efficiency and maintenance of cellular reprogramming. In addition to the Company’s core programs, the broad potential of both the MOGRIFY and epiMOGRIFY technologies allow continued exploration and generation of novel cell-based and in vivo reprogramming therapies for disease areas with high unmet clinical need.

Mogrify also announced the appointments of Dr Lorenz Mayr, former CTO of GE Healthcare Life Sciences and Vice President Discovery Sciences at AstraZeneca, as Non-Executive Director, after serving as an Observer since July 2020, and Dr Hiromichi Kimura, Astellas Venture Management as an Observer.

Dr. Darrin M. Disley, OBE, CEO, Mogrify, said: "We are delighted to see the continued support of our existing investors, as well as welcome new investors Astellas Venture Management and Dr Jonathan Milner, in support of the progression of our initial programs through to IND-enabling studies.

Their commitment is a testament to our delivery of world-class science, expertise in cellular reprogramming and the unique potential of the MOGRIFY and epiMOGRIFY platforms to develop novel in vivo reprogramming therapies while also addressing issues of scalability and manufacturing in ex vivo cell therapies. This latest funding will provide runway toward a significant Series B round as we progress our lead assets toward the clinic."

Alastair Kilgour, Chief Investment Officer, Parkwalk Advisors, said: "Mogrify is led by a team with proven world-class commercial and scientific expertise who have made significant progress in applying the technology to drive the speed, efficiency and maintenance of cellular reprogramming. We are excited to continue supporting their extraordinary team as the Company progresses with increasing success in cell and gene therapy development."

For further information about investing in Mogrify, please visit: View Source