US FDA approval tracker: May

 The most valuable approval of the year was bestowed in May − that of Lilly’s Mounjaro in type 2 diabetes. According to Evaluate Pharma its consensus 2028 forecasts are over $8bn, and the product is gearing up to take market share from Novo’s diabetes franchise. Elsewhere it was good news for Dupixent as the anti-IL-4/1L-13 MAb became the first FDA-approved drug for the chronic inflammatory disease eosinophilic oesophagitis (EoE). The decision for Sanofi/Regeneron’s product came over two months early. Competing data from Astrazeneca’s phase 3 Messina study testing the anti-IL-5 MAb Fasenra in EoE are expected in the second half of the year. It was not all good news last month: UCB and Verrica both received knockbacks, while Avadel, Amicus and Pfizer/Myovant all had their Pdufas extended. On the theme of delays, TG Therapeutics became the latest to suffer a three-month extension, announcing yesterday that the Pdufa for ublituximab in relapsing multiple sclerosis had been pushed out to the end of December. This came after a long list of setbacks for the company.

Notable first-time US approval decisions in May
Project	Company	Indication(s)	2028e sales by indication (SBI) ($m)	Outcome
Mounjaro (tirzepatide)	Lilly	Type 2 diabetes	8,132	Approved
Bimzelx	UCB	Plaque psoriasis	1,191	CRL (pre-approval inspections)
AXS-05	Axsome	Major depressive disorder	787	Pending (Q2)
FT218	Avadel	Treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy	361	Full approval not expected until mid-2023, when Jazz's '963 REMS patent expires
VP-102	Verrica	Molluscum contagiosum	359	CRL (deficiencies at CMO)
Miglustat	Amicus	Pompe disease	266*	Delayed to Aug 29 (cipaglucosidase Pdufa also delayed to Oct 29, combination product known as AT-GAA)
Vtama (tapinarof)	Dermavant	Plaque psoriasis	-	Approved
Annik (penpulimab)	Akeso/Sino	3L nasopharyngeal carcinoma	-	Pending (H1)
Voquenza triple pak, Voquenza dual pak (vonoprazan)	Phathom	Adults with H pylori infection	-	Approved
Radicava ORS (MT-1186, oral edaravone)	Mitsubishi Tanabe	ALS	-	Approved
Lamotrigine (ET-105) for supspension	Eton/Azurity	Epilepsy	-	CRL
*Forecasts for AT-GAA. Source: Evaluate Pharma & company releases.

 

Supplementary and other notable approval decisions in May
Product	Company	Indication (clinical trial)	Outcome
Myfembree	Pfizer/Myovant	Moderate to severe pain associated with endometriosis (Spirit 1, Spirit 2)	Delayed to Aug 6 (FDA needs more time to review bone mineral density information)
Tyvaso DPI	Mannkind/United Therapeutics	PAH and pulmonary hypertension-associated interstitial lung disease	Approved
Evrysdi	Roche/PTC	Pre-symptomatic infants under 2 months old with spinal muscular atrophy (Rainbowfish)	Approved
Opdivo + Yervoy, Opdivo + chemo	Bristol Myers Squibb	1L unresected advanced, recurrent or metastatic oesophageal squamous cell carcinoma (Checkmate-648)	Approved
Enhertu	Astra/Daiichi	Her2+ve breast cancer after anti-Her2-therapy (Destiny-Breast 03)	Approved
Olumiant	Lilly	Treatment of certain hospitalised patients with Covid-19	Approved
Kymriah	Novartis	3L follicular lymphoma (ph2 Elara)	Approved
Tpoxx IV	Siga	Smallpox	Approved
Dupixent	Sanofi/Regeneron	Eosinophilic oesophagitis, 12 years and older (NCT03633617)	Approved (over 2mth early)
Annovera	Therapeutics MD	Birth control, revisions to in vitro release testing specification	Approved
Vidaza	Bristol Myers Squibb	Paediatric patients with newly diagnosed juvenile myelomonocytic leukaemia (AZA-JMML-001)	Approved
Tibsovo + Vidaza	Servier	Newly diagnosed IDH1-mutated AML in adults 75 years or older (Agile)	Approved
Fylnetra (Neulasta biosimilar)	Amneal	Neutropenia	Approved
Source: Evaluate Pharma & company releases.

 

FDA Covid-19 EUAs
Product	Company	Outcome
Janssen Covid-19 vaccine  (Ad26.COV2-S)	J&J	Authorised use limited to individuals aged 18+ for whom other authorised or approved vaccines are not accessible or clinically appropriate, and to individuals aged 18+ who elect to receive the J&J vaccine because they would otherwise not receive a vaccine (received EUA in Feb 21)
Comirnaty	Pfizer/ Biontech	Single booster dose authorised for individuals aged 5-11
Source: company releases. https://www.evaluate.com/vantage/articles/insights/nme-approvals-snippets/us-fda-approval-tracker-may

Immunic misses primary endpoint in Phase 2 ulcerative colitis tria;

 Trial Did Not Achieve Primary Endpoint in the Ulcerative Colitis (UC) Population Caused by Unexpected Interference Between Vidofludimus Calcium and Concurrent Use of Corticosteroids –

– In UC Population Without Concurrent Steroid Use, Pooled Vidofludimus Calcium Data Suggest Activity in Clinical Remission Over Placebo; Counterbalanced by Interference Observed in the UC Population with Concurrent Steroid Use –

– Company Does Not Plan Further Development Activities in Ulcerative Colitis Without a Partner –

– Focus to Remain on Ongoing Phase 3 Development of Vidofludimus Calcium in Multiple Sclerosis, and Ongoing IMU-935 and IMU-856 Programs with Clinical Data for Both Expected in 2022 –

– $93.1 Million in Cash and Cash Equivalents as of May 31, 2022 Expected to Fund Immunic
Into the Fourth Quarter of 2023 –

– Conference Call and Webcast, Including a Corporate Update, to be Held today,
June 2, 2022 at 8:00 am ET –

Immunic will host a webcast today at 8:00 am ET. To participate in the webcast, please register in advance at: https://imux.zoom.us/webinar/register/WN_XgdotnImTHKxQiLFa958mA or on the "Events and Presentations" section of Immunic's website at: ir.imux.com/events-and-presentations. Registrants will receive a confirmation email containing a link for online participation or a telephone number for dial in access.

An archived replay of the webcast will be available approximately one hour after completion on Immunic's website at: ir.imux.com/events-and-presentations.

https://finance.yahoo.com/news/immunic-inc-reports-top-line-103000663.html

Aeglea Bio Gets Refusal to File Lette r from FDA

 Aeglea BioTherapeutics, Inc. (NASDAQ: AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics to benefit people with rare metabolic diseases, today announced that it received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) regarding the company's Biologics License Application (BLA) for pegzilarginase for the treatment of Arginase 1 Deficiency (ARG1-D).

In the RTF letter the FDA requested additional data to support effectiveness, such as evidence showing that plasma arginine and metabolite reduction predicts clinical benefit in patients with ARG1-D or clinical data demonstrating a treatment effect on clinically meaningful outcomes. The FDA also requested additional information relating to Chemistry Manufacturing and Controls (CMC). There were no issues related to safety raised in the letter. Aeglea intends to request a Type A meeting with the FDA to clarify and respond to the items identified in the RTF letter.

https://finance.yahoo.com/news/aeglea-biotherapeutics-receives-refusal-file-125900337.html

Zosano Pharma Files Voluntary Petition for Relief Under Chapter 11

 Zosano Pharma Corporation (NASDAQ:ZSAN), a clinical-stage biopharmaceutical company, today announced that on June 1, 2022 it filed a voluntary petition for relief under chapter 11 of title 11 (“Chapter 11”) of the United States Bankruptcy Code (the “Bankruptcy Code”) in the United States Bankruptcy Court for the District of Delaware (the “Bankruptcy Court”), thereby commencing a Chapter 11 case for the company.

Zosano continues to operate its business as a “debtor-in-possession” under the jurisdiction of the Bankruptcy Court and in accordance with the applicable provisions of the Bankruptcy Code and orders of the Bankruptcy Court. Zosano is seeking approval of a variety of “first day” motions containing customary relief intended to enable the company to continue its ordinary course operations and to facilitate an orderly wind down of its operations. The company intends to sell substantially all of its assets during the bankruptcy case.

Additional information about the Chapter 11 case, including access to Bankruptcy Court documents, is available online at http://www.kccllc.net/ZosanoPharma, a website administered by KCC, a third-party bankruptcy claims and noticing agent.

https://finance.yahoo.com/news/zosano-pharma-files-voluntary-petition-120000029.html

PDS Gets Fast Track Designation for head-neck cancer combo

 PDS Biotech has been granted Fast Track designation for PDS0101 in combination with KEYTRUDA® (pembrolizumab) for the treatment of recurrent or metastatic HPV16-positive head and neck cancer

https://finance.yahoo.com/news/pds-biotechnology-granted-fda-fast-120000127.html

Yumanity Therapeutics Sees Large Growth in Short Interest

 Yumanity Therapeutics, Inc. (NASDAQ:YMTX – Get Rating) was the recipient of a significant increase in short interest in the month of May. As of May 15th, there was short interest totalling 138,000 shares, an increase of 25.5% from the April 30th total of 110,000 shares. Approximately 1.7% of the company’s shares are sold short. Based on an average daily trading volume, of 416,000 shares, the short-interest ratio is currently 0.3 days.

https://www.defenseworld.net/2022/06/01/yumanity-therapeutics-inc-nasdaqymtx-sees-large-growth-in-short-interest.html

Repare in Worldwide License and Collaboration Agreement with Roche for Camonsertib

 Repare will receive a $125 million upfront payment and is eligible to receive up to an additional $1.2 billion in potential development, regulatory, commercial and sales milestones, plus royalties on global net product sales

Repare to host conference call today at 5:00 p.m. EDT

The Company will host a conference call with accompanying slides for analysts and investors today at 5:00 p.m. Eastern Time to further discuss the collaboration. To access the call, please dial (877) 870-4263 (U.S.) or (855) 669-9657 (Canada) or (412) 317-0790 (international) at least 10 minutes prior to the start time and ask to be joined to the Repare Therapeutics call. A live video webcast will be available in the Investor section of the Company’s website at https://ir.reparerx.com/news-and-events/events. A webcast replay will also be archived for at least 30 days.

https://finance.yahoo.com/news/repare-therapeutics-announces-worldwide-license-200500192.html