On August 9, 2021 Mission Therapeutics ("Mission"), a drug discovery and development company focused on targeting the ubiquitin pathway, and AbbVie ("AbbVie")(NYSE: ABBV) reported progression of selected deubiquitylating enzyme (DUB) targets into the next phase of research in their neurodegenerative disease collaboration (Press release, Mission Bio, AUG 9, 2021, View Source [SID1234586152]).

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In the second major milestone of the Companies’ research and preclinical development collaboration, AbbVie has nominated two DUB targets to progress to the next stage of drug discovery. This selection follows supportive data from in vitro and in vivo Alzheimer’s and Parkinson’s disease models. As a result of the nomination, Mission will receive a milestone payment of $20 million from AbbVie.

Alzheimer’s and Parkinson’s diseases are the most common neurological disorders worldwide, with over 50 million people living with dementia and Alzheimer’s disease, and 10 million more with Parkinson’s. While there are treatments to help reduce symptoms, there are no treatments available to help stop or reverse progression.

Both Alzheimer’s and Parkinson’s are associated with the accumulation of misfolded, proteins in the brain, which are believed to cause impaired function and death of nerve cells. DUBs can help to maintain cell health by regulating the degradation of these proteins.

Under the terms of the agreement, first announced in 2018, Mission and AbbVie are collaborating to identify novel DUB targets and discover associated inhibitor compounds for neurodegenerative diseases. AbbVie has the option to gain exclusive rights to develop and commercialize inhibitors of selected DUB targets and will pay Mission success-based milestones as well as royalty payments for each commercialized product.

Commenting on the development, Dr Anker Lundemose, Mission’s CEO, said:

"We are delighted to have reached this next major milestone in our collaboration with AbbVie and receive the $20 million milestone – this represents a big step forward for Mission and further highlights DUBs as targets for drug development. The successful and timely progression of two DUB targets into the drug discovery phase is further validation of our platform. We look forward to the continuation of this great neurodegenerative disease collaboration."

Dr Eric Karran, Vice President, Neuroscience Discovery Research, AbbVie commented:

"There is an urgent need for treatments that can make tangible and lasting improvements to the lives of Alzheimer’s and Parkinson’s patients. Our collaboration with Mission has the potential to identify novel therapeutic options for neurodegenerative disorders. We have had a great experience working with Mission and are pleased to be able to continue to draw on their valued expertise as we enter the next phase of drug development."

On August 9, 2021 Adaptimmune Therapeutics plc (Nasdaq: ADAP), a leader in cell therapy to treat cancer, reported financial results for the second quarter ended June 30, 2021 and provided a business update (Press release, Adaptimmune, AUG 9, 2021, View Source [SID1234586120]).

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"With the data presented at ASCO (Free ASCO Whitepaper) for afami-cel and our planned BLA filing next year, I am pleased with progress on our ‘2-2-5-2’ strategy," said Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer. "We will present data updates from our Phase 1 trials, SURPASS and ADP-A2AFP, at upcoming conferences and I am confident that Adaptimmune is well-placed to maintain its leadership position in cell therapies for cancer."

Upcoming confirmed data updates
ADP-A2AFP Phase 1 Trial at ILCA

On Sunday, September 5, 2021, Dr. Bruno Sangro of Clinica Universidad de Navarra will present data from Cohort 3 and the expansion phase of the Phase 1 trial of ADP-A2AFP in liver cancer during an oral presentation scheduled for 12:25 p.m. CET at the International Liver Cancer Association’s (ILCA) Annual Conference
As of the April 5th data cut-off used for ILCA, 13 patients had received ADP-A2AFP in Cohort 3 and expansion, 11 patients were evaluable with at least one post-baseline scan
SURPASS Phase 1 Trial at ESMO (Free ESMO Whitepaper)

The Company will present an update from its Phase 1 SURPASS trial in an e-poster at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) congress that will be available online September 16th
As of the August 2nd data cut-off, 25 patients had received ADP-A2M4CD8, 23 patients had at least one post-baseline scan
The trial continues to recruit in lung, gastroesophageal, head and neck, and bladder cancers – focus indications, for which there have been early signs of efficacy, including responses, with SPEAR T-cells targeting MAGE-A4
Based on early data from patients with ovarian cancer treated in the trial, the Company is planning to add this as a focus indication to the SURPASS trial
SPEARHEAD-1 and afami-cel BLA

As presented at ASCO (Free ASCO Whitepaper) 2021, the Company reported an overall response rate for patients with at least one scan (evaluated by RECIST 1.1 per investigator assessment) of 39.3% (13/33), 41.4% (12/29) for synovial sarcoma, including two complete responses, and 25.0% (1/4) for MRCLS from its Phase 2 SPEARHEAD-1 trial
Of the 29 patients with synovial sarcoma, the disease control rate (defined as either response or stable disease) was 86.2% (25/29 patients) with 2 complete responses and 10 partial responses
This data set is intended to support planned BLA filing next year
Next data update planned at CTOS 2021
The European Medicines Agency and the FDA have agreed to Adaptimmune’s pediatric investigational plans
Working with key industry leaders to prepare for a successful commercial launch
Adaptimmune has partnered with Agilent for the development, manufacturing, and supply of a companion diagnostic for the MAGE-A4 biomarker
The Company is also working with Miltenyi Biotec for the process validation, manufacture, and supply of the lentiviral vector for use in the product for commercial launch
Further indications for next-gen SPEAR T-cell therapies
SURPASS-2

Planning to initiate a Phase 2 clinical trial with ADP-A2M4CD8, SURPASS-2, in esophageal and esophagogastric junction cancers in the third quarter of 2021
Other early-stage programs

The Company ceased enrollment in the Radiation Sub-Study of the afami-cel Phase 1 trial at the end of July. Five patients were treated in this sub-study and the Company plans to provide a data update at an upcoming congress
Corporate

The Company received a development milestone payment of $4.2 million in the three months ended June 30, 2021
Financial Results for the three and six months ended June 30, 2021

Cash / liquidity position: As of June 30, 2021, Adaptimmune had cash and cash equivalents of $50.5 million and Total Liquidity1 of $285.4 million.
Revenue: Revenue for the three and six months ended June 30, 2021 was $3.1 million and $3.5 million, respectively, compared to $0.5 million and $1.3 million for the same periods in 2020. Revenue has increased primarily due to an increase in development activities under the Astellas Collaboration Agreement.
Research and development (R&D) expenses: R&D expenses for the three and six months ended June 30, 2021 were $28.9 million and $53.4 million, respectively, compared to $20.5 million and $41.7 million for the same periods in 2020. R&D expenses increased due to an increase in the number of employees engaged in research and development, and increases in costs related to the development of a companion diagnostic assay and our Phase 2 clinical trial associated with ADP-A2M4CD8. These increases were partially offset by an increase in reimbursements receivable for research and development tax and expenditure credits.
General and administrative (G&A) expenses: G&A expenses for the three and six months ended June 30, 2021 were $13.5 million and $27.4 million, respectively, compared to $10.3 million and $19.6 million for the same periods in 2020 due to increases in employee-related costs, share-based compensation expense, and professional fees.
Net loss: Net loss attributable to holders of the Company’s ordinary shares for the three and six months ended June 30, 2021 was $39.1 million and $76.8 million respectively ($(0.04) and $(0.08) per ordinary share), compared to $29.9 million and $58.0 million ($(0.04) and $(0.07) per ordinary share) for the same periods in 2020.
Financial Guidance

The Company believes that its existing cash, cash equivalents and marketable securities will fund the Company’s current operations into early 2023, as further detailed in the Company’s Quarterly Report on Form 10-Q for the three and six months ended June 30, 2021, to be filed with the Securities and Exchange Commission following this earnings release.

Conference Call Information
The Company will host a live teleconference and webcast to provide additional details at 4:30 p.m. EDT (9:30 p.m. BST) today. A live webcast of the conference call and replay can be accessed at https://bit.ly/3zFas59. An archive will be available after the call at the same address. To participate in the live conference call, if preferred, please dial (833) 652-5917 (US or Canada) or +1 (430) 775-1624 (International). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (7867634).

On August 9, 2021 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported the receipt of notification from its partner, AbbVie Inc., regarding its termination of the license and collaboration agreement for the investigational drug abicipar pegol for the treatment of neovascular age-related macular degeneration (nAMD) and Diabetic Macular Edema (DME) (Press release, Molecular Partners, AUG 9, 2021, View Source [SID1234586136]). As such, Molecular Partners will regain the development and commercial rights of abicipar on a worldwide basis.

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"There remains a significant unmet medical need for patients living with nAMD and DME, and we remain confident in abicipar’s potential to offer these patients a differentiated treatment option over existing therapies," said Patrick Amstutz, Chief Executive Officer of Molecular Partners. "Our focus for this program will be determining the best path to value creation within the context of our expansive portfolio of antiviral and immuno-oncology therapies in development."

Molecular Partners will form a special committee to evaluate the program and determine appropriate next steps. In addition, Molecular Partners and AbbVie will continue their ongoing discovery alliance, in which AbbVie will continue to evaluate additional DARPin candidates for ophthalmic indications. The return of the abicipar program is not expected to impact Molecular Partners’ financial outlook for 2021 or previously issued guidance.

Abicipar is a long-acting anti-VEGF DARPin molecule which was invented by Molecular Partners and initially licensed to Allergan in 2011. The program has been through two positive Phase 3 studies, CEDAR and SEQUOIA, which supported the non-inferior efficacy of the abicipar quarterly dosing regimen to maintain vision gains with more than 50 percent fewer injections versus ranibizumab (13 vs. 6) dosed monthly in the first year.

With the acquisition of Allergan by AbbVie, the rights to abicipar were transferred to AbbVie. In June 2020, AbbVie received a Complete Response Letter to the Biologics License Application for abicipar pegol, indicating that the rate of intraocular inflammation observed following administration of Abicipar pegol resulted in an unfavorable benefit-risk ratio in the treatment of nAMD (AMD), and that additional work would be required to demonstrate a lower rate of ocular inflammation than what was previously seen in the Phase 3 studies.

On August 9, 2021 Sierra Oncology, Inc. (NASDAQ: SRRA), a late-stage biopharmaceutical company on a mission to deliver targeted therapies that treat rare forms of cancer, reported guidance on the development timeline for its lead product candidate, momelotinib (Press release, Sierra Oncology, AUG 9, 2021, View Source [SID1234586153]). The company now expects topline data from the Phase 3 registration-enabling MOMENTUM study by February 2022, and assuming positive results, the company plans to file a New Drug Application with the U.S. Food & Drug Administration in Q2 2022.

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Sierra also announced it has raised $34 million in gross proceeds thus far in Q3 2021 through the use of its at-the-market (ATM) offering. These funds are in addition to the $90.7 million in cash and cash equivalents on hand at the close of the second quarter of 2021, further bolstering the company’s cash balance as it prepares for momelotinib commercialization. The company also has the potential to obtain up to $33.3 million in cash from Series B warrants that must be exercised within 75 days of the announcement of MOMENTUM topline data.

Stephen Dilly, MBBS, PhD, President and Chief Executive Officer of Sierra Oncology noted, "The continued excellent execution of the MOMENTUM study and further acceleration of the timeline, combined with the additional funding secured over the last few weeks, puts us in a very strong position as we approach topline data."

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