bluebird bio Enters into Advance Agreement to Sell Priority Review Voucher, if Granted, for $103 Million

On October 30, 2023 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported that it has entered into an agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV), if received, in connection with the potential approval of lovotibeglogene autotemcel (lovo-cel) for sickle cell disease (Press release, bluebird bio, OCT 30, 2023, View Source [SID1234636432]). Under the terms of the agreement, rights to the PRV will transfer to the buyer and the Company will receive $103 million upon closing of the sale, which is contingent upon the U.S. Food and Drug Administration’s (FDA’s) approval of the biologics license application (BLA) for lovo-cel and granting of the PRV.

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The Company’s BLA for lovo-cel was previously accepted for priority review by the FDA for patients with sickle cell disease ages 12 and older who have a history of vaso-occlusive events (VOEs) and has a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2023. bluebird may be eligible for a PRV should lovo-cel be approved for patients under the age of 18.

"The potential sale of a priority review voucher would provide an important source of non-dilutive capital for bluebird ahead of the anticipated launch of lovo-cel," said Chris Krawtschuk, chief financial officer, bluebird bio. "As the FDA completes its review of lovo-cel, our team remains confident in the robustness and maturity of our BLA package for individuals 12 and older with sickle cell disease and looks forward to a regulatory decision by the end of this year."

Closing of the transaction remains subject to the approval of lovo-cel and receipt of a PRV from the FDA, as well as customary closing conditions.

About the Rare Pediatric Disease Priority Review Voucher Program

The Rare Pediatric Disease Priority Review Voucher Program is intended to encourage development of new drug and biological products for the prevention and treatment of certain rare pediatric diseases. Under this program, upon approval, the US Food and Drug Administration awards priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. The voucher can be redeemed to receive priority review of a subsequent marketing application for a different product. PRVs may be sold or transferred, and there is no limit on the number of times a PRV can be transferred.

Biomea Fusion Reports Third Quarter 2023 Financial Results and Corporate Highlights

On October 30, 2023 Biomea Fusion, Inc. ("Biomea" or "the Company") (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to discovering and developing novel covalent small molecules to treat and improve the lives of patients with genetically defined cancers and metabolic diseases, reported third quarter 2023 financial results and business highlights (Press release, Biomea Fusion, OCT 30, 2023, View Source [SID1234636431]).

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"With the disease modifying potential demonstrated by BMF-219, we are excited to begin the expansion portion of COVALENT-111 in type 2 diabetes, and to initiate the evaluation of BMF-219’s impact in type 1 diabetes with our COVALENT-112 study. Both studies are now open for enrollment more than a quarter ahead of schedule. With the potential to enable patients to retain and regenerate their own insulin-producing beta cells, BMF-219 may represent a promising new approach, if ultimately approved. We believe BMF-219 has the potential to halt and reverse disease progression for people with both type 1 and type 2 diabetes, enabling them to improve glycemic control while being off therapy and avoid insulin dependency," said Thomas Butler, CEO and Chairman of Biomea. "In the third quarter, we also announced the first clinical data of BMF-219 in relapsed/refractory AML patients with menin-dependent mutations, which we believe yield encouraging efficacy and safety data. Finally in this quarter, we also initiated the clinical study of our second, Biomea-discovered investigational covalent inhibitor, BMF-500, a novel FLT3 inhibitor. We look forward to reporting on our pipeline progress in both diabetes and oncology in the upcoming quarters."

THIRD QUARTER 2023 CLINICAL AND REGULATORY HIGHLIGHTS

DIABETES

COVALENT-111 (BMF-219 for Type 2 Diabetes)
BMF-219 is an investigational diabetes therapy that in an early clinical trial has shown promising blood glucose control in adults with uncontrolled type 2 diabetes at Week 12, after 4 weeks dosing and 8 weeks off treatment.
During the escalation phase of COVALENT-111, a total of 32 type 2 diabetes patients completed 4-weeks of dosing with BMF-219 to date (10 active patients per arm, with dose levels 100 mg with food, 100 mg, 200 mg and 200 mg with food (n=2)). Compared to baseline, 84% of all patients dosed for four weeks with BMF-219 showed a reduction in HbA1c at Week 4 and 74% at Week 12 (n=32), two months after the final dose of BMF-219. During the 4-week dosing period, BMF-219 was generally well tolerated; there were no dose reductions, dose discontinuations, or severe or serious adverse events. Also, during the off-treatment period, no severe or serious treatment emergent adverse events were noted.
FDA and Health Canada cleared the initiation of the expansion portion of COVALENT-111, which will evaluate BMF-219 administered at 100 mg and 200 mg, with dosing durations up to 12 weeks in approximately 200 type 2 diabetes patients.
Anticipated Upcoming Milestones:
Report topline data for the escalation portion in Q4 2023 to include multiple BMF-219 dosing cohorts (n=10 per cohort: 50 mg, 100 mg, 200 mg, 100 mg BID, 100 mg with food, and 200 mg with food (n=2)).
Start enrollment of expansion portion of COVALENT-111 at two dose levels including 100 mg and 200 mg, with a fourth cohort following the completion of the escalation portion.
COVALENT-112 (BMF-219 for Type 1 Diabetes)
BMF-219 has shown the potential to specifically regenerate and retain insulin-producing beta cells in preclinical studies.
Announced FDA clearance of IND for Phase II study COVALENT-112 of BMF-219 in type 1 diabetes. The study is designed to enroll 150 adults with type 1 diabetes and examine the safety and efficacy of BMF-219 at two oral dose levels, 100 mg and 200 mg, for 12 weeks of treatment followed by a 40 week off-treatment period.
Anticipated Upcoming Milestones:
Dose the first patient in COVALENT-112.
ONCOLOGY

COVALENT-101 (BMF-219 for Oncology)
Reported initial topline data from ongoing Phase I clinical trial (COVALENT-101) which showcased initial responses in relapsed/refractory AML patients with menin-dependent mutations.
New data revealed 2 CRs out of 5 pretreated patients with relapsed/refractory AML patients carrying menin-dependent mutations (MLL1r, NMP1, MLL1-PTD, and NUP98 fusion) treated at Dose Level 4.
BMF-219, the first and only investigational covalent small-molecule menin inhibitor in clinical development, was generally well tolerated with no dose-limiting toxicities observed, and no related QTc prolongation reported.
Continued site activation and patient enrollment to establish optimal dose levels across four liquid tumor cohorts including patients with AML/ALL, DLBCL, MM and CLL.
Anticipated Upcoming Milestones:
Report additional details of the clinical data set of AML/ALL patients dosed in the COVALENT-101 study at an upcoming medical conference.
COVALENT-102 (BMF-219 for KRAS-Mutant Solid Tumors)
BMF-219 is the first investigational menin inhibitor in clinical development for the treatment of solid tumors; as a pan-KRAS inhibitor BMF-219 is under evaluation in subsets of NSCLC, CRC, and PDAC patients.
Continued site activation and patient enrollment to establish optimal dose levels across all three solid tumor indications (NSCLC, CRC and PDAC with an activating KRAS mutation).
COVALENT-103 (BMF-500 for Acute Leukemias)
Dosed first patient with BMF-500, a novel investigational third generation oral covalent inhibitor of FLT3 and the second product candidate discovered and developed by Biomea’s proprietary FUSION System.
FUSIONTM SYSTEM PLATFORM / ONGOING EFFORTS

Continued to advance development candidates derived from Biomea’s proprietary FUSION System platform to discover novel covalently binding small molecules. Both BMF-219 and BMF-500 were discovered via the FUSION System, each within 18 months from target identification to IND candidate selection.
THIRD QUARTER 2023 FINANCIAL RESULTS

Cash, Cash Equivalents, Restricted Cash, and Investments: As of September 30, 2023, the Company had cash, cash equivalents, restricted cash, and investments of $199.5 million, compared to $113.4 million as of December 31, 2022.
Net Income/Loss: Biomea reported a net loss attributable to common stockholders of $28.4 million for the three months ended September 30, 2023, compared to a net loss of $22.9 million for the same period in 2022. Net loss attributable to common stockholders was $82.4 million for the nine months ended September 30, 2023, compared to a net loss of $56.5 million for the same period in 2022.
Research and Development (R&D) Expenses: R&D expenses were $25.3 million for the three months ended September 30, 2023, compared to $18.2 million for the same period in 2022. The increase of $7.1 million was primarily due to an increase clinical development cost and external consulting related to the Company’s product candidates, BMF-219 and BMF-500, as well as increase in personnel-related costs. R&D expenses were $71.7 million for the nine months ended September 30, 2023 compared to $42.2 million for the same period in 2022. The increase of $29.5 million was primarily due to an increase personnel-related costs as well as an increase in clinical development and manufacturing costs related to the Company’s product candidates, BMF-219 and BMF-500.
General and Administrative (G&A) Expenses: G&A expenses were $5.8 million for the three months ended September 30, 2023, compared to $5.2 million for the same period in 2022. G&A expenses were $17.1 million for the nine months ended September 30, 2023 compared to $15.2 million for the same period in 2022. The increase in both periods was primarily due to increased personnel-related expenses, including stock-based compensation, due to an increase in headcount.

Bicycle Therapeutics to Participate in Upcoming Investor Conferences

On October 30, 2023 Bicycle Therapeutics plc (NASDAQ:BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported that management will participate in the following investor conferences in November (Press release, Bicycle Therapeutics, OCT 30, 2023, View Source [SID1234636430]):

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TD Cowen 7th Annual Fall Oncology Innovation Summit on Friday, November 3, 2023; fireside chat at 10:00 a.m. ET
Jefferies London Healthcare Conference on Wednesday, November 15, 2023; fireside chat at 11:00 a.m. ET

Live webcasts of the fireside chats will be accessible in the Investors section of Bicycle’s website at www.bicycletherapeutics.com. Archived replays of the webcasts will be available following the fireside chat dates.

Entry into a Material Definitive Agreement

On October 30, 2023, Athersys, Inc. (the "Company") reported to have entered into an offer letter (the "Letter Agreement") with a holder ("Holder") of the Company’s common stock purchase warrants issued on October 16, 2023 (the "Existing Warrants") to (1) reduce the exercise price of the Existing Warrants from $0.1395 to $0.01 per share (the "Reduced Exercise Price"), (2) issue to the Holder new common stock purchase warrants (the "New Warrants") to purchase up to 20,000,000 shares of the Company’s common stock at an exercise price of $0.01 per share; and (3) make the Existing Warrants immediately exercisable (Filing, 8-K, Athersys, OCT 30, 2023, View Source [SID1234636429]).

Pursuant to the Letter Agreement, the Holder agreed to (A) reduce the period of the subsequent equity sale restrictions contained in Section h)(i) of Annex A to the warrant exercise inducement offer letter dated October 11, 2023 between the Holder and the Company (the "October 11, 2023 Letter") from 60 days to 30 days; (B) reduce the applicable period in each and all of the provisions relating to restrictions on the Company (or any of its subsidiaries) involving a "Variable Rate Transaction" (as such term, or a term of similar meaning, is defined in the applicable document) so that each such period terminates on March 1, 2024 in all stock purchase agreements, related letter agreements, and all other related agreements with the Holder; and (C) vote as set forth below in connection with a Fundamental Transaction.

The Holder agreed that upon the occurrence of a Fundamental Transaction (as defined in the New Warrants), if the Holder holds any shares of the Company’s common stock and a stockholder vote is required for such Fundamental Transaction, then the Holder agrees to vote all shares of its common stock that it holds as of any such record date that may be taken the vote of stockholders that are entitled to vote on such Fundamental Transaction in accordance with the recommendations of the Company’s board of directors.

We also agreed to file a registration statement (the "Registration Statement") on Form S-1 (or other appropriate form) providing for the resale of the shares of common stock issuable upon exercise of the New Warrants (the "New Warrant Shares"), on or before November 12, 2023, and to use commercially reasonable efforts to have such Resale Registration Statement declared effective by the U.S. Securities and Exchange Commission (the "SEC") within 90 days following the date of filing with the SEC and to keep the Resale Registration Statement effective at all times until no holder of the New Warrants owns any New Warrants or New Warrant Shares.

In addition, on October 30, 2023, the Company and the Holder entered into an Amendment to Series A Common Stock Purchase Warrants (the "Warrant Amendment") which amended the Existing Warrants to reflect (i) the Reduced Exercise Price, (ii) that the Existing Warrants are now immediately exercisable, and (iii) that the Existing Warrants will terminate on October 30, 2028. The Warrant Amendment is filed as Exhibit 10.2 to this Current Report on Form 8-K and is incorporated herein by reference. The foregoing description of the Warrant Amendment is qualified in its entirety by reference to such exhibit.

New Warrant Terms

The following summary of certain terms and provisions of the New Warrants is not complete and is subject to, and qualified in its entirety by, the provisions of the New Warrants, the form of which is filed as Exhibit 4.1 to this Current Report on Form 8-K and is incorporated herein by reference. The following description of the New Warrants is qualified in its entirety by reference to such exhibit.

Exercise Period and Dilution

Each New Warrant will have an exercise price equal to its applicable Reduced Exercise Price. The New Warrants are immediately exercisable on the date of issuance and may be exercised for a period of five years therefrom. The exercise price and number of shares of common stock issuable upon exercise is subject to appropriate proportional adjustment in the event of share dividends, share splits, reorganizations or similar events affecting our common stock and the exercise price.

Exercisability

The New Warrants will be exercisable, at the option of each holder, in whole or in part, by delivering to us a duly executed exercise notice and, within the earlier of (i) two trading days and (ii) the number of trading days comprising the standard settlement period with respect to the common stock as in effect on the date of delivery of the notice of exercise thereafter, payment in full for the number of shares of common stock purchased upon such exercise (except in the case of a cashless exercise as discussed below). A holder may not exercise any portion of the New Warrant to the extent that the holder, together with its affiliates and any other persons acting as a group together with any such persons, would own more than 4.99% (or, at the election of the purchaser, 9.99%) of the number of shares of common stock outstanding immediately after exercise (the "Beneficial Ownership Limitation"); provided that a holder with a Beneficial Ownership Limitation of 4.99%, upon notice to us and effective  61 days after the date such notice is delivered to us, may increase the Beneficial Ownership Limitation so long as it in no event exceeds 9.99% of the number of shares of common stock outstanding immediately after exercise.

Cashless Exercise

If, at the time a holder exercises its New Warrants, a registration statement registering the issuance of the shares of common stock underlying the New Warrants under the Securities Act is not then effective or available for the issuance of such shares, then in lieu of making the cash payment otherwise contemplated to be made to us upon such exercise in payment of the aggregate exercise price, the holder may only exercise its New Warrants (either in whole or in part), at such time by means of a cashless exercise in which the holder shall be entitled to receive upon such exercise the net number of shares of common stock determined according to a formula set forth in the New Warrants, which generally provides for a number of shares of common stock equal to (A) (1) the volume weighted average price on (x) the trading day preceding the notice of exercise, if the notice of exercise is executed and delivered on a day that is not a trading day or prior to the opening of "regular trading hours" on a trading day or (y) the trading day of the notice of exercise, if the notice of exercise is executed and delivered after the close of "regular trading hours" on such trading day, or (2) the bid price on the day of the notice of exercise, if the notice of exercise is executed during "regular trading hours" on a trading day and is delivered within two hours thereafter, less (B) the exercise price, multiplied by (C) the number of shares of common stock the New Warrant was exercisable into, with such product then divided by the number determined under clause (A) in this sentence.

Fractional Shares

No fractional shares of common stock will be issued upon the exercise of the New Warrants. Rather, we will, at our election, and in lieu of the issuance of such fractional share, either (i) pay cash in an amount equal to such fraction multiplied by the exercise price or (ii) round up to the next whole share issuable upon exercise of the New Warrant.

Transferability

Subject to applicable laws, a New Warrant may be transferred at the option of the holder upon surrender of the New Warrant to us together with the appropriate instruments of transfer and funds sufficient to pay any transfer taxes payable upon such transfer.

Market

There is no trading market available for the New Warrants on any securities exchange or nationally recognized trading system. We do not intend to list the New Warrants on any securities exchange or nationally recognized trading system. The shares of common stock issuable upon exercise of the New Warrants are currently quoted on the Pink Open Market tier operated by OTC Markets Group, Inc. (the "OTC Pink") under the symbol "ATHX."

Rights as a Stockholder

Except as otherwise provided in the New Warrants or by virtue of such holder’s ownership of shares of the Company’s common stock, the holders of the New Warrants do not have the rights or privileges of holders of the Company’s common stock, including any voting rights, until they exercise their New Warrants.

Fundamental Transaction

In the event of a fundamental transaction, as described in the New Warrants and generally including any reorganization, recapitalization or reclassification of our common stock, the sale, transfer or other disposition of all or substantially all of our properties or assets, our consolidation or merger with or into another person, the acquisition of more than 50% of our outstanding shares of common stock, the holders of the New Warrants will be entitled to receive upon exercise of the New Warrants the kind and amount of securities, cash or other property that the holders would have received had they exercised the New Warrants immediately prior to such fundamental transaction. Additionally, in the event of a fundamental transaction, we or any successor entity will, at the option of the holder of a New Warrant exercisable at any time concurrently with or within 30 days after the consummation of the fundamental transaction (or, if later, the date of the public announcement thereof), purchase the New Warrant from the holder by paying to the holder an amount of consideration equal to the value of the remaining unexercised portion of such New Warrant on the date of consummation of the fundamental transaction based on the Black-Scholes option pricing model, determined pursuant to a formula set forth in the New Warrants. The consideration paid to the holder will be the same type or form of consideration that was offered and paid to the holders of shares of common stock in connection with the fundamental transaction; provided that if no such consideration was offered or paid, the holders of common stock will be deemed to have received common stock of the successor entity in such fundamental transaction for purposes of this provision of the New Warrants.

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ArriVent Receives FDA Breakthrough Therapy Designation for Furmonertinib for First-Line Treatment of Advanced or Metastatic Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations

On October 30, 2023 ArriVent Biopharma, Inc., a clinical-stage company dedicated to accelerating the global development of innovative biopharmaceutical therapeutics, reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for furmonertinib for the treatment of patients with previously untreated, locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations (Press release, ArriVent Biopharma, OCT 30, 2023, https://arrivent.com/arrivent-receives-fda-breakthrough-therapy-designation-for-furmonertinib-for-first-line-treatment-of-advanced-or-metastatic-non-small-cell-lung-cancer-with-egfr-exon-20-insertion-mutations/?utm_source=rss&utm_medium=rss&utm_campaign=arrivent-receives-fda-breakthrough-therapy-designation-for-furmonertinib-for-first-line-treatment-of-advanced-or-metastatic-non-small-cell-lung-cancer-with-egfr-exon-20-insertion-mutations [SID1234636428]).

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"Breakthrough Therapy designation is an important step forward in our development of furmonertinib and highlights its exciting potential as a first-line therapy for patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations," said Bing Yao, Chairman, Co-founder and Chief Executive Officer of ArriVent.

Stuart Lutzker, Co-founder and President of R&D added: "This FDA designation underscores the encouraging clinical activity we have seen with furmonertinib in the FAVOUR study and reflects the critical need for effective and tolerable therapeutic options for these patients. We look forward to continuing our work with the agency as we progress our furmonertinib clinical development program in NSCLC, including our ongoing pivotal, global Phase 3 FURVENT trial evaluating furmonertinib in previously untreated NSCLC patients whose tumors contain EGFR exon 20 insertion mutations."

The Breakthrough Therapy designation was granted based on interim results from FAVOUR trial (NCT04858958), a Phase 1b, randomized, open-label, multi-center clinical trial evaluating the efficacy and safety of furmonertinib in patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations. FDA’s Breakthrough Therapy designation is designed to expedite development and review of drugs intended to treat a serious or life-threatening condition for which preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapies. Interim results from the trial demonstrated furmonertinib has promising anti-tumor activity as a single agent with a well-tolerated safety profile in the first-line and previously treated patients. The pivotal Phase 3 FURVENT trial (NCT05607550) of furmonertinib for the treatment of first-line NSCLC with EGFR exon 20 insertion mutations is currently enrolling patients globally.